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Rare Myopathy Affects Women, Too, Advocate Emphasizes
After being diagnosed as a carrier of x-linked myotubular myopathy (XLMTM) and losing her infant ...
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Blood Pressure Medicine Combo Helps Slow Heart Decline in DMD Study
A treatment combination of two blood pressure medicines can help slow heart function decline in ... -
From ‘Motor Moron’ to a Limb-Girdle MD Diagnosis
As I noted in previous columns, everyone is different. You are who the other is ... -
Partnership Aims to Lower Out-of-Pocket Costs for Rare Disease Meds
AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology ... -
DMD Trial Testing Oral ASP0367 Recruiting Boys 8–16 in US
A Phase 1b clinical trial evaluating Astellas Pharma’s ASP0367 is recruiting boys with Duchenne muscular dystrophy ... -
How to Enjoy Travel With Duchenne Muscular Dystrophy
Last week, I traveled to the headquarters of my employer, BioNews, the publisher of Muscular ... -
The Excitement and Fear That Come With Giving Your Child a Sibling
Most weeks, I toss around column ideas with my friends and family, asking them for ... -
New MRI Technique Provides Whole-body Analyses in FSHD Trial
AMRA Medical’s new MRI method was found to play an instrumental role in detecting changes ... -
The Effects of Guilt on My Body and Mind
Welcome back to “Hidden Truths.” This week I wanted to move away from the style ... -
Nominations Open for 2022 Eurordis Black Pearl Awards
Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. ... -
Long-term Viltepso Prevents Motor Function Decline in DMD Boys
Long-term treatment with Viltepso (viltolarsen) safely and effectively prevents a decline in motor function in ... -
Seeking Discomfort Can Help Me Accomplish What Seems Impossible
I recently started watching a YouTube channel called Yes Theory, which inspires people to “seek ... -
Givinostat Appears to Slow Muscle Decline in Becker MD Trial
Givinostat halted muscle shrinkage and fat infiltration in men with Becker muscular dystrophy (BMD) despite ... -
LION-101 Gene Therapy for Rare MD Type Now on FDA Fast Track
The U.S. Food and Drug Administration (FDA) has granted fast track designation to LION-101, an ... -
‘A Space in Time’ Captures Joys, Struggles of Growing Up With DMD
“Can some of them get better, Daddy?” a young boy asks his father on the ... -
Centogene Sets Mission to Cure 100 Rare Diseases in Next 10 Years
At its first virtual investor event, biotech company Centogene set a bold mission: to cure ... -
Losmapimod Fails at FSHD Trial’s Primary Goal, But Shows Potential
Oral losmapimod, an investigational treatment for people with facioscapulohumeral muscular dystrophy (FSHD), showed clinically relevant ... -
Caring for My Son Helps Me Continue Being a Lifelong Learner
Before my 2-year-old son, Alfie, was born, I was a primary school teacher. While I ... -
Vita Raises $32M to Advance Cell Therapies for LGMD, Other Dystrophies
Vita Therapeutics announced that it has raised $32 million in financing to advance its work into ... -
The Power and Importance of Thought
What does it mean to live? Different people and places may prompt varied answers to ... -
CureDuchenne Link Invites MD Patients, Carriers to Help in Research
CureDuchenne has launched the first comprehensive data hub to connect biosamples and clinical and genetic ... -
EveryLife Introduces First of Kind ‘Roadmap’ to ICD Codes
To help patient advocacy leaders and their partners better understand how global health statistics codes ... -
What to Do When You Feel Overwhelmed
My life has been quite busy lately. Between starting my full-time job at BioNews, helping ... -
Get Ready for NORD’s Patient and Family Forum, Set for June 26-27
Those who wish to gain practical tools for living optimally with rare diseases are encouraged ... -
It’s Year 15 for Jared Sweet Golf Outing DMD Fundraiser
The 15th Annual Jared Sweet Golf Outing, a Michigan event that benefits Parent Project Muscular ... -
Cholesterol Metabolism Is Potential Therapeutic Target in DMD
An analysis of microRNA molecules isolated from the blood of people with Duchenne muscular dystrophy ... -
Despite My Initial Fears, I Now Praise the Feeding Tube
Last June, my 2-year-old son, Alfie, had his first surgery to place a feeding tube. It ... -
DMD’s Course With Corticosteroid Use Examined in Review Study
With current corticosteroid use, people with Duchenne muscular dystrophy (DMD) typically lose their walking ability ... -
PACER Panic: Remembering the Awkward Days of Gym Class
I stood behind my father in uncomfortable silence. The summer had come to a close, and ... -
Who Are You?: Welcome to ‘Hidden Truths’
How would you respond if I asked, “Who are you?” While the question may seem ... -
Ionis Molecule Shows Early Promise for Myotonic Dystrophy Type 1
High levels of the toxic RNA molecule associated with myotonic dystrophy type 1 (DM1) lead to ... -
Rare 2030 Action Campaign Pushing for European Plan for Rare Diseases
Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European ... -
Facing a New Series of Physical Transitions
Transitions are a part of life. We go through puberty as teenagers and our bodies ... -
Advocates Can Try for Grants Ahead of Rare Disease Week on Capitol Hill
Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the ... -
Vamorolone Safely Improves Muscle Function in DMD Boys, Trial Finds
Top-line data from the VISION-DMD Phase 2b clinical trial evaluating vamorolone in boys with Duchenne muscular ... -
CRISPR Gene Editing Therapy Is Promising in New Mixed Model
A CRISPR-Cas9-based gene editing therapy promoted the production of a smaller but functional version of ... -
Grieving the Challenges That Lie Ahead for My Son
Last week, I took my 2-year-old son, Alfie, for a walk-through at a local preschool. ... -
Gene Therapy Trial for Rare Form of MD Planned for Next Year
Asklepios BioPharmaceutical (AskBio) is planning to launch a Phase 1/2 clinical study to evaluate LION-101, ... -
Using Cheat Codes to Live My Best Life With Becker Muscular Dystrophy
Up, up, down, down, left, right, left, right, B, A, Start. Cheat codes were a ... -
1st DMD Mouse Model to Mimic Heart Disease Confirms Gene Therapy Efficacy
For the first time, a new mouse model has been shown to mimic the heart ... -
Writing About Rare Disease for a Living Is Rewarding
On April 1, I started full time at BioNews, the publisher of Muscular Dystrophy News ... -
DMD Gene Therapy, SRP-9001, Showing ‘Robust’ Micro-dystrophin in Trial
Sarepta Therapeutics‘ investigational gene therapy SRP-9001 showed early signs of biologic efficacy in muscle biopsies ... -
Two More Patients Dosed in Solid Trial of DMD Gene Therapy SGT-001
Two more participants have been dosed in the IGNITE DMD clinical trial, bringing to eight ... -
Helping My Son Conserve Energy Is a Constant Juggling Act
Growing up, I was never good at performing tricks. I couldn’t whistle, I’ve never been ... -
FDA Puts Investigational FSHD Therapy Losmapimod on Fast Track
The U.S. Food and Drug Administration (FDA) has granted fast track designation to losmapimod, a ... -
Jesse’s Journey Supports 4 Duchenne Projects, Including Gene Editing
Jesse’s Journey, a Canadian nonprofit founded by parents of a child with Duchenne muscular dystrophy ... -
Imagining a Life Without Duchenne Muscular Dystrophy
I sometimes like to imagine a life in which I don’t have Duchenne muscular dystrophy, ... -
CIFFREO Trial of Potential DMD Gene Therapy Delayed in US
Pfizer has announced a delay in opening U.S. sites for its Phase 3 clinical trial ... -
Positive Results Reported From Phase 2 Study of SRP-5051 in DMD
Sarepta Therapeutics has announced positive results from a global study evaluating its investigational treatment SRP-5051 ... -
Couple with FSHD Relish Shared Independence, Advocacy Work
Ray Jordan and Cyndi Segroves, a married couple with facioscapulohumeral muscular dystrophy (FSHD), haven’t let ... -
After Falling, I Pick Myself Up and Bend My Armor Back Into Shape
The glint of armor demands attention. The jousting lance was slung to the side of ... -
New Tool Shows Profound Metabolic Changes in DMD Patients’ Muscles
The muscles of people with Duchenne muscular dystrophy (DMD) show profound impairment, with changes in ... -
Why It’s So Important to Find Your People
When Alfie was first diagnosed, I went searching. I needed answers. I needed to know ... -
#AANAM – Presenter Compares Features of DM1 and DM2
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 Virtual AAN ... -
#AANAM – Pregnancy May Worsen Muscle Weakness in LGMDR9 Patients
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 Virtual ... -
Quick Approval for COVID Vaccines a Concern, Rare Disease Patients Say
More than half of the people with rare diseases and their caregivers, asked in a ... -
#AANAM – Corticosteroids Aid Muscle Mass in BMD and LGMD, Trials Needed
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 Virtual AAN ... -
#AANAM – Investigational DM1 Therapy Shows Promise
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How I’ve Managed Constipation as Someone With Duchenne
This is the dirtiest column I have ever written because it’s about poop. And whether ... -
EveryLife Scholarship Fund Open Until May 7 for Adults With Rare Diseases in US
The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to ... -
DuchenneXchange Launches Mobile, Desktop App for DMD Community
The DuchenneXchange online social platform has launched a mobile and desktop app for the Duchenne muscular dystrophy ... -
FDA Places Pamrevlumab for DMD on Fast Track
The U.S. Food and Drug Administration has granted fast track designation to pamrevlumab, an investigational ... -
Keisha Greaves Named MDA’s 2021 National Goodwill Ambassador
The Muscular Dystrophy Association (MDA) has named Keisha Greaves, a fashion entrepreneur and neuromuscular disease advocate ... -
Audentes Becomes Astellas Gene Therapies, Will Advance MD Treatments
Following its acquisition last year by Astellas Pharma, Audentes Therapeutics now has become Astellas Gene ... -
Dungeons and Dragons Helps Me Live Without Duchenne for a Moment
Kriki Cragwalker, a redheaded dwarf from the mountain city of Jagarz Dauk, grasps her enchanted, ... -
Flavocoxid Well-tolerated, Lessens Inflammation in Small DMD Trial
Flavocoxid was well-tolerated and lessened inflammation in a small clinical trial involving boys with Duchenne ... -
Cost of Rare Diseases in US? Nearly $1 Trillion in 2019, EveryLife Foundation Finds
As anyone affected by a rare disease knows, treating the illness while trying to go ... -
What a ‘Typical’ Day Is Like for My Son, Alfie, and Me
BEEP BEEP BEEP BEEP. My eyes spring open. It’s still dark. It’s not my alarm ... -
Second Phase 3 Study of Pamrevlumab Recruiting Boys With DMD
FibroGen has begun a second Phase 3 clinical trial of pamrevlumab, given with corticosteroids, among ... -
#MDA2021 – Long-term Vamorolone Slowed Disease Progression in DMD Boys
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual ... -
#MDA2021 – DMD Gene Therapy PF-06939926 Continues to Show Promise
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual ... -
#MDA2021 – Gene Therapy SGT-001 Shows Continued Benefit in DMD Boys
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual ... -
#MDA2021 – Factors Likely to Predict Wheelchair Use With FSHD1 Detailed
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual ... -
#MDA2021 – LGMD2E Gene Therapy SRP-9003 Shows Benefits After 2 Years
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual ... -
What Having Duchenne Muscular Dystrophy Feels Like
If you are familiar with the disease Duchenne muscular dystrophy, it’s easy to tell if ... -
#MDA2021 – Plasma Exchange Enables Effective Gene Therapy Redosing
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual ... -
#MDA2021 — Gene Therapy for DMD Exon Duplication Showing Promise in Trial
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual ... -
#MDA2021 – Patients, Families Discuss Diagnosis, Symptoms of LGMD2i
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual ... -
#MDA2021 – SRP-5051 Shows Promise in Animal Studies, Supporting Phase 2 Trial
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual ... -
Springtime Brings Renewed Opportunity for Activity and Adventure
It’s been a sedentary, stale winter in the northern U.S. Spring is afoot, and I ... -
#MDA2021 – Pandemic Added Access Challenges for Patients Surveyed Globally
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual ... -
#MDA2021 — Amondys 45 Safe and Well-tolerated in Phase 1/2 Trial
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual ... -
#MDA2021 — Patients Switched to Emflaza for Efficacy, Tolerability
Editor’s note: The Muscular Dystrophy News Today team is providing in-depth coverage of the 2021 MDA Virtual ... -
What I’ve Learned Since My Son’s LAMA2 MD Diagnosis
On Oct. 21, 2019, I sat in a doctor’s office with my 11-month-old son, Alfie, ... -
MDA Engage Offers Free Educational Events
Through a program called MDA Engage, the Muscular Dystrophy Association (MDA) is offering virtual educational ... -
#MDA2021 – MDA Virtual Conference to Feature Latest in Clinical Care, Research
Next week’s 2021 MDA Virtual Clinical and Scientific Conference is expected to attract more than 1,200 clinicians ... -
I’m Ready to Go Forward After My 2nd Dose of the COVID-19 Vaccine
On Feb. 19, I received my second dose of the Pfizer COVID-19 vaccine. According to ... -
Pink, Green, Blue and Purple Lights Illuminated World for Rare Disease Day
Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is ... -
Vamorolone Trial for DMD Finishes Pivotal First Part, Results Awaited
The final patient has finished the first, six-month portion of the pivotal VISION-DMD Phase 2b ... -
A Hefty Guitar Impacts My Ability to Keep the Music Going
When I was a kid, music from the likes of Led Zeppelin, Fleetwood Mac, and ... -
Dosing Begins in Phase 2 Trial Testing Oral Therapy BBP-418 for LGMD2i
The first patient has been dosed in a Phase 2 trial testing BBP-418, a potential ... -
Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion
Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of ... -
Exon Skipping Dominates DMD Treatment, But What’s on Horizon?
With the recent approval of Amondys 45 (casimersen) by Sarepta Therapeutics, there are now four exon-skipping ... -
FDA Approves Amondys 45 for Duchenne Patients With Exon 45 Mutation
The U.S. Food and Drug Administration (FDA) has conditionally approved Amondys 45 — formerly known as casimersen — by ... -
Givinostat Continues to Delay DMD Progression in Long-term Study, New Data Show
Treatment with givinostat alongside corticosteroids continues to delay disease progression — and maintain patients’ ability ... -
Rare Disease Day at NIH, Set for March 1, Growing Year by Year
Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place ... -
Yes, We Are Rare, but Please Realize We Are Human First
Rare Disease Day, a global push for rare disease awareness, is less than a week ... -
Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, ... -
MDA Launches 39th Annual #Shamrocks Fundraiser
The Muscular Dystrophy Association (MDA) has launched its 39th annual #MDAShamrocks campaign — the largest ... -
How an Albino Direwolf Named Ghost Made Me Think About Being Rare
“A white wolf in a white wood, silent as a shadow.” – “A Dance with ... -
Deleting Mss51 Gene Reduces Fatigue, Increases Endurance in DMD Mice
By deleting the Mss51 gene, scientists were able to reduce fatigue, increase endurance, and ease ... -
PPMD Receives Award to Improve Neurobehavioral Care in Duchenne
Parent Project Muscular Dystrophy (PPMD) has received an award of more than $100,000 from the ... -
Pandemic Won’t Stop Rare Disease Day on Feb. 28
Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 ... -
Translarna Increased Dystrophin Levels in Phase 2 DMD Study
Treatment with Translarna (ataluren) increased levels of the protein dystrophin in young boys with Duchenne ... -
It’s Time for Me to Go Outdoors and Take in the Vitamin D
In the last year, it’s been easy for me to coop myself up in my ... -
Isometric Exercise in DMD Found to Improve Boys’ Strength, Function
Mild to moderate isometric leg exercise — contractions of a particular muscle or group of ... -
NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns
While progress was made last year on newborn screening and other policy issues critical to ... -
Welcome to the Castle: Facing Down and Defeating Your Fears
His palms firmly pressed against the pommel, he holds the sword close to his chest. ... -
New App Plays ‘Pivotal’ Role in Increasing Registry Successes
In the first year since Parent Project Muscular Dystrophy (PPMD) and Thread launched a mobile ... -
STRIVE Applications for Duchenne Community Now Open
Applications are now open for the seventh annual Strategies to Realize Innovation, Vision, and Empowerment ... -
PPMD Invests $1M in Satellos With Goal of Repairing Muscle Cells in Duchenne
Parent Project Muscular Dystrophy (PPMD) announced a $1 million investment in Satellos Bioscience to support ... -
Why I’m Taking a Break From Playing Video Games
I’m a self-described gamer, and proud of it, but lately I’ve realized I’ve probably been ... -
New CureDuchenne Initiative Provides Support, Education on COVID-19 Vaccination
CureDuchenne has launched a new initiative to prioritize the needs of the Duchenne muscular dystrophy (DMD) ... -
A Bittersweet Farewell to My Column Readers
Dear readers, This will be my last column for Muscular Dystrophy News. I want to ... -
Exon 45 Skipping Therapy for DMD Shows Safety in Small Trial
A potential exon-skipping therapy for Duchenne muscular dystrophy (DMD) called DS-5141 showed a good safety profile with ... -
Dosing Begins in Phase 3 Trial of Pfizer’s Gene Therapy for Duchenne
The first boy has been dosed in a Phase 3 trial testing Pfizer‘s gene therapy, PF-06939926, ... -
I Make Sure My Support System Is Strong
When I’m deep in thought, reflecting on my life, I shudder to think who and ... -
DMD Boys Experience Biological, Functional Gains in SRP-9001 Study
The investigational gene therapy SRP-9001 safely leads to the production of a functional form of ... -
Why Freelancing Works for My Life With Duchenne
Since my internship at The Washington Post, I’ve done freelance work at outlets ranging from ... -
Pivotal Trial Begins for AMO-02 in Steinert Disease
AMO Pharma has announced the initiation of REACH-CDM, a pivotal Phase 2/3 clinical trial that ... -
Top 10 Muscular Dystrophy Stories of 2020
Throughout 2020, Muscular Dystrophy News Today brought you daily coverage of important discoveries, treatment advancements, ... -
Edgewise Will Use $95M Funding to Advance EDG-5506 for BMD, DMD
Edgewise Therapeutics has received $95 million in funding to advance the clinical development of its ... -
It’s Time to Spread Some Holiday Cheer
Christmas is upon us, and for many, it might not even feel like the holiday ... -
Genethon Cleared to Launch French Trial Testing GNT0004 Gene Therapy for DMD
The French National Agency for Medicines and Health Products Safety has cleared Généthon to begin ... -
Lower-dose SRP-5051 Potentially More Effective Than Exondys 51, Phase 2 Trial Finds
Sarepta Therapeutics’ investigational treatment SRP-5051 enhanced cell penetration, exon skipping, and dystrophin levels at a ... -
Translarna Approved in Russia for DMD Patients 2 and Older
Translarna (ataluren) has been approved in Russia for treating Duchenne muscular dystrophy (DMD) caused by nonsense ... -
Lilly, Precision to Develop Gene Editing Therapies for DMD, Other Disorders
A new collaboration between Eli Lilly and Precision BioSciences aims to develop new gene editing ... -
Give Yourself Some Peace of Mind This Holiday Season
This year has been full of seemingly insurmountable struggles for everyone. As we resolved one ... -
How to Avoid a Fall
Having Duchenne muscular dystrophy is scary for a number of reasons. High up on that ... -
MDA Wants Neuromuscular Disease Patients to Have Early Access to COVID-19 Vaccine
The Muscular Dystrophy Association (MDA) is encouraging the U.S. Centers for Disease Control and Prevention ... -
ReveraGen Receives $3.3M NIH Grant to Advance Vamorolone in DMD
ReveraGen BioPharma has been awarded a $3.3 million grant from the National Institute of Neurological ... -
Sarepta Talks Progress, Plans for New Trials of DMD Gene Therapy SRP-9001
Sarepta Therapeutics is about to launch a Phase 1 trial of SRP-9001, its investigational gene ... -
Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe
The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and ... -
Looking at Holiday Self-isolation in a Positive Light
The holidays are notorious for being one of the most hectic times of the year. ... -
DM1 Patients Asked to Join Natural History Study Aiming for Better Clinical Trials
The Myotonic Dystrophy Clinical Research Network (DMCRN) is asking adults with myotonic dystrophy type 1 ... -
Expressing Our Negative Emotions to Find Positive Ones
I always hope to impart a positive message to my readers and exude joy in ... -
Phase 1 Trial of Oral EDG-5506 for Becker MD Enrolling at Texas Site
Edgewise Therapeutics announced the opening of a Phase 1 clinical trial to assess the safety ... -
Q&A: Meet the Muscular Dystrophy Association’s New CEO and President
The Muscular Dystrophy Association (MDA) announced last week it had selected former MDA vice chairman ... -
MDA Launches Tool to Make Clinical Data More Accessible to Researchers
The Muscular Dystrophy Association (MDA) and DNAnexus have introduced a digital platform to improve patient care ... -
Saying Goodbye to Eye Strain, Thanks to Blue-light Glasses
Technology is an essential tool I use both in my social life and to be ... -
AMO-02 for Congenital DM1, Entering Pivotal Trial, Gets Rare Pediatric Disease Status
The U.S. Food and Drug Administration (FDA) has given rare pediatric disease status to AMO Pharma‘s investigational ... -
How I’m Getting Out of My 2020 Funk
If 2020 hasn’t been a challenging year for you, that’s great news. But for me, ... -
Ultragenyx and Solid Biosciences Partner on DMD Gene Therapy
Ultragenyx Pharmaceutical and Solid Biosciences announced a strategic collaboration to develop and commercialize new gene ... -
PPMD Seeking Adults with Duchenne or Becker MD to Join Advisory Committee
Parent Project Muscular Dystrophy (PPMD) is seeking adults with Duchenne or Becker muscular dystrophy to ... -
Phase 3 Trial Failure Prompts Catabasis to Stop Edasalonexent Development
Catabasis Pharmaceuticals’ investigational oral therapy edasalonexent failed to preserve muscle function and overall functionality in boys ... -
Long-term Corticosteroid Use Delays Lung and Heart Decline in DMD Boys, Study Finds
Long-term corticosteroid treatment delays respiratory decline and heart disease in boys with Duchenne muscular dystrophy (DMD) regardless of daily ... -
Rapper Wheelz Hosts Oct. 24 ‘Ryan’s Day’ Virtual Fundraiser for DMD
The 11th annual Ryan’s Day fundraiser, organized by rapper Wheelz to raise awareness of Duchenne ... -
The 4 Categories of Tiredness
Those of us with invisible chronic illnesses master the art of mindfulness for our bodies. ... -
MDA Telethon Returning, Going Virtual With Host Kevin Hart
The Muscular Dystrophy Association Telethon returns this month after a six-year absence, with actor Kevin ... -
What I Learned About Life From My Dog
On Sept. 29, part of my life was lost. My dog, Gus, who had been ... -
Edasalonexent Helps to Preserve Heart and Bone Health, New Data Show
Edasalonexent, a potential oral therapy for Duchenne muscular dystrophy (DMD), can prevent the development of ... -
Phase 3 Trial of Puldysa Stopped for Lack of Efficacy in Duchenne Patients
Santhera Pharmaceuticals has decided to stop SIDEROS, its Phase 3 trial assessing the effectiveness of ... -
Pfizer’s Gene Therapy for Duchenne Placed on Fast Track by FDA
The U.S. Food and Drug Administration (FDA) has granted fast track designation to PF-06939926, Pfizer’s ... -
FDA Lifts Clinical Hold on IGNITE DMD Gene Therapy Trial
The U.S. Food and Drug Administration (FDA) has lifted its clinical hold on the Phase ... -
Living for Myself While Hoping for the Right Person
I have clear memories of listening to my fourth-grade friends talk about what their “boyfriends” ... -
Removing Barriers to Employment for People With Disabilities
As a journalist and person with Duchenne muscular dystrophy, I have a dual purpose. I ... -
Durable Safety and Motor Gains Evident in Trial of SRP-9001, DMD Gene Therapy
A single dose of SRP-9001, Sarepta Therapeutics’ investigational gene therapy for Duchenne muscular dystrophy (DMD), continues to ... -
Givinostat Wins Rare Pediatric Disease Designation; Phase 3 DMD Trial Fully Enrolled
Italfarmaco has now completed recruitment — delayed in the spring due to the COVID-19 pandemic — ... -
Vamorolone Continues to Improve Motor Function in DMD Boys, Phase 2 Trials Show
The experimental therapy vamorolone can improve motor function significantly in boys with Duchenne muscular dystrophy (DMD), without stunting ... -
Muscular Dystrophy and Other Clinical Trials Adapting to Life With COVID-19
In a Facebook Live event hosted by the Muscular Dystrophy Association (MDA), experts discussed the ... -
The Value of Having Friends With the Same Disease
Over the last couple of years, I’ve had opportunities to meet and befriend more adults ... -
Real-world Data Could Replace Placebo Groups in DMD Clinical Trials, Study Indicates
Real-world and natural history data could be used to supplement, or perhaps replace, the use ... -
Sarepta Poised to Start Next Trial of SRP-9001 for Duchenne MD
Sarepta Therapeutics has completed a meeting with the Office of Tissues and Advanced Therapies (OTAT) ... -
Overcoming the Idea of Being a Burden
As I’ve started relying more on people for things I used to do on my ... -
Differences in Key Proteins in DMD Muscle Cells Favor Fat Formation, Study Says
An imbalance in cells producing proteins called aldehyde dehydrogenases (ALDHs) — favoring fat formation in ... -
Santhera Acquires Global Rights to DMD Therapy Vamorolone
Santhera Pharmaceuticals has obtained the worldwide rights to vamorolone (VBP15), an experimental therapy for Duchenne ... -
September is Duchenne Action Month
September is Duchenne Action Month, an awareness, fund-raising, and educational campaign supporting people with Duchenne ... -
Virtual Events Mark MDA’s Muscular Dystrophy Awareness Month
This year, the Muscular Dystrophy Association (MDA) will celebrate MDA National Muscular Dystrophy Awareness Month, held ... -
Casimersen, Exon 45 Skipping Duchenne Therapy, Under FDA Priority Review
An application requesting the approval of casimersen (SRP-4045) to treat Duchenne muscular dystrophy (DMD) amenable to exon 45 ... -
The Battle Against Ableism Is Tiring, but I Won’t Give Up
A few unfortunate circumstances recently have left me mentally exhausted by the endless recurrence of ... -
Viltepso Commercially Available in the US for DMD Treatment
NS Pharma‘s Viltepso (viltolarsen) is now commercially available in the U.S. for people with Duchenne ... -
Why It’s Important to Be Still Amid the World’s Chaos
The world can be overwhelming thanks to the deadly coronavirus pandemic, especially for someone dealing ... -
First Patients Enroll in Phase 1 Trial of EPM-01, Potential Becker MD Therapy
A Phase 1 clinical trial of EPM-01 ((+)-epicatechin), a potential oral treatment of Becker muscular dystrophy that ... -
Phase 3 Trial of Pamrevlumab Enrolling DMD Patients Unable to Walk
The LELANTOS Phase 3 clinical trial, which is evaluating FibroGen‘s pamrevlumab in people with Duchenne ... -
EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US
The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to ... -
FDA Approves Viltolarsen, Now Viltepso, for DMD Patients With Exon 53 Mutation
The U.S. Food and Drug Administration (FDA) has conditionally approved NS Pharma‘s Viltepso (viltolarsen) for ... -
I’m Sleeping Comfortably Now, Thanks to a New Mattress
Most mitochondrial myopathy patients rely on sleep as therapy for our disease. Yes, rest can ... -
Patient Feedback Being Sought on Puldysa for Possible NHS Funding
The National Institute for Health and Care Excellence (NICE) is urging patient organizations to gather ... -
Losing My Ability to Walk Won’t Stop Me
When I was younger, I couldn’t imagine losing my ability to get up from the ... -
MD Advocates Offer Advice for Students With Neuromuscular Disorders Amid COVID-19
The importance of communication and self-care for people with muscular dystrophy and other neuromuscular diseases ... -
FDA Requests More Data, Maintains Clinical Hold on IGNITE DMD Trial of SGT-001 Gene Therapy
The U.S. Food and Drug Administration (FDA) is maintaining the clinical hold on the Phase 1/2 trial ... -
The Angels Who’ve Profoundly Affected My Life
Living with a disability often means I’m exposed to people’s true colors. This can be ... -
A Day on the Hill: Using Our Voices to Lobby Lawmakers
Columnist’s Note: Dear reader, this will be my final column at Muscular Dystrophy News. The ... -
FDA Places SRP-9001, DMD Micro-dystrophin Gene Therapy, on Fast Track
An investigational gene therapy for Duchenne muscular dystrophy (DMD), called SRP-9001 micro-dystrophin, was given fast track designation ... -
Stem Cell Therapy VTA-110 Named FDA Orphan Drug for DMD Treatment
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Vita Therapeutics‘ ... -
Benitec Begins Early Study of BB-301 Gene Therapy for OPMD
Benitec Biopharma has begun the first study to support the start of clinical trials of ... -
Two Imaging Biomarkers Predict Walking Ability in DMD, Useful for Trials
Two magnetic resonance measures — MRI and magnetic resonance spectroscopy (MRS) — of leg muscles are ... -
A Good Sense of Humor Makes my Duchenne Diagnosis Easier
I like to think I’m pretty funny, at least that is until I work up ... -
NS Pharma Opens Support Hub to Help DMD Patients Access Treatments
NS Pharma has launched a support hub, called NS Support, for people in the U.S. with ... -
Hansa Licenses Imlifidase to Sarepta as Gene Therapy Pre-treatment for DMD, LGMD
Hansa Biopharma has granted an exclusive worldwide license to Sarepta Therapeutics to develop and promote ... -
Being Independent Within My Capabilities
I often associate independence with adulthood and coming-of-age. The liberation of a teenager learning to ... -
National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US
The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease ... -
UK Renews Puldysa Early Access Program for Duchenne Patients
Oral Puldysa (idebenone), Santhera Pharmaceuticals‘ investigational treatment for breathing problems caused by Duchenne muscular dystrophy ... -
FDA Asked to Approve Casimersen, Exon 45 Skipping DMD Therapy
Sarepta Therapeutics has submitted a full application to the U.S. Food and Drug Administration (FDA) ... -
Don’t Feel Bad About Calling Me ‘Inspiring’
In the disabled community, many people discuss the negative implications of being called “inspiring.” Some ... -
Coronavirus Relief Fund Created to Help DMD Patients, Families
The COVID-19 pandemic has forced many to shelter at home, caused millions of layoffs, and ... -
CHMP Recommends Translarna Label Update That Enables Treating DMD Patients Unable to Walk
The Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency, ... -
Looking Backward but Living Life Forward With MD
“Life can only be understood backwards; but it must be lived forwards,” said philosopher Søren ... -
Phase 1 Trial of Potential Oral Treatment for LGMD2i Doses 1st Volunteer
A Phase 1 clinical trial of BBP-418, a potential first oral therapy for limb-girdle muscular dystrophy ... -
Dating in the Era of Coronavirus
It is undeniable that the coronavirus pandemic has affected many aspects of life. Along with ... -
Collaboration Provides Cough Assist Machines to British Columbia Residents With Neuromuscular Disease
A partnership between Muscular Dystrophy Canada (MDC) and Technology for Living (TFL) will seek to ... -
Creativity Allows Me to Surpass Any Physical Limits
For a long time, I didn’t think journalism was a creative endeavor. I viewed the ... -
Sarepta’s Gene Therapy for DMD Showing to Safety, Efficacy at One Year
At the one year mark, Sarepta Therapeutics’ SRP-9001 micro-dystrophin gene therapy continues to show positive ... -
MDA Holds Q&A on Navigating COVID-19, and Area Reopenings, Safely
People with neuromuscular disorders such as muscular dystrophy are not at a higher risk of ... -
Duchenne Has Forced Me to Reflect on My Own Mortality
My parents tried to shield me as long as they could from data that revealed ... -
Viltolarsen Earns European Orphan Drug Designation as DMD Treatment
The European Commission has granted orphan drug designation to viltolarsen, a treatment for people with ... -
Medicare’s Role for People with Disabilities
On May 1, I became eligible for Medicare. This is one of those milestones in ... -
SRP-9003 Gene Therapy for LGMD2E Shows Benefits After 1 Year
A single administration of the gene therapy SRP-9003 at a low dose continues to lead ... -
Learning to Accept My Mental Illness Through Creativity
Positivity is a characteristic that I naturally possess. I seldom struggle to find the bright ... -
Santhera and Rutgers Partner on Gene Therapy Work for LAMA2 MD
Santhera Pharmaceuticals announced the signing of two agreements with Rutgers University related to the development of ... -
$1M Gene Therapy Research Grant Launched by PPMD, Duchenne UK
Parent Project Muscular Dystrophy (PPMD) and Duchenne UK have joined forces to offer up to ... -
What I’ve Learned from Starting a Twitch Channel
When I started my Twitch channel in February, I wasn’t sure what to expect. I ... -
Let’s Aim to Achieve True Global Accessibility
On May 21, the world celebrated its ninth Global Accessibility Awareness Day, but accessibility awareness ... -
Viltolarsen Raises Dystrophin Levels, Aids Muscles in DMD Boys in Phase 2 Trial
NS Pharma’s lead candidate viltolarsen safely and effectively increases dystrophin levels and promotes improvements in boys with Duchenne ... -
Trial Supports ATL1102’s Safety in Improving Muscle Strength, Function in DMD Boys
A Phase 2 trial of ATL1102 met its primary goal of demonstrating favorable safety and tolerability ... -
SIDEROS Phase 3 Trial of Puldysa in Duchenne Patients Completes Enrollment
The SIDEROS Phase 3 clinical trial, investigating if oral Puldysa (idebenone) can be added to a stable glucocorticoid ... -
Pfizer’s DMD Gene Therapy Continues to Show Promise, Phase 1b Trial Shows
Pfizer’s experimental gene therapy PF-06939926 promotes sustained production of a shorter but functional version of ... -
CAP-1002 Improves Muscle, Lung and Heart Function in Young DMD Patients, HOPE-2 Trial Data Show
One-year treatment with CAP-1002, Capricor Therapeutics’ investigational cell therapy for Duchenne muscular dystrophy (DMD) significantly improves respiratory, cardiac ... -
Why Self-monitoring Our Functioning Levels Is Important
The muscular dystrophy world is ever-evolving. I began to have symptoms of muscle loss and ... -
IGNITE DMD Trial Remains on Hold, FDA Requests More Gene Therapy Data
The U.S. Food and Drug Administration (FDA) is requesting more information on the process used ... -
Living Joyfully Helped Me Overcome Fear, Anger, and Sadness
Joy is one of the best tools we have to combat the progressive loss of ... -
Experts Offer Advice for Duchenne, Becker Patients Amid COVID-19 Pandemic
People with Duchenne and Becker muscular dystrophy should not forego their routine health assessments during the COVID-19 pandemic, but ... -
The Power of Empathy Among Friends
I’ve never considered myself to be insecure about my disability. My use of a mobility ... -
CureDuchenne Ventures Gives $1M to Support Non-viral Gene Therapy Work
CureDuchenne Ventures has given $1 million in seed funding to support the development of Myosana Therapeutics‘ ... -
PTC and Sarepta Say MD Treatment Supplies Uninterrupted, Encourage Use of Patient Support Programs
Both PTC Therapeutics and Sarepta Therapeutics expect to continue providing an uninterrupted supply of their Duchenne ... -
The Impossibility of Putting a Value on a Life
Author’s note: The COVID-19 crisis has already lasted several months. The reality is that we ... -
Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say
While there are few silver linings to the cloud created by COVID-19, the pandemic that ... -
MDA’s Medical Advisory Team to Help Guide Research and Patient Care
The Muscular Dystrophy Association (MDA) has formed a Medical Advisory Team to provide guidance related ... -
COVID-19 Delays Phase 3 Trial of Givinostat in DMD, Italfarmaco Says in Webinar
Because of the global COVID-19 pandemic, Italfarmaco has paused enrollment of boys with Duchenne muscular ... -
The ‘Crip Camp’ Utopia Made Us a Better Society Today
Netflix’s new documentary “Crip Camp” transports viewers to the peak era of peace-preaching hippies and ... -
Duchenne MD Patient Tanner Rico Approaches Life With Optimism
Traci and Tony Rico of Cardiff-by-the-Sea, California, juggle more than their share of family health ... -
How to Best Live with One Another During Quarantine
COVID-19 has forced families around the world to stay indoors and spend a lot more ... -
Living, Learning, Thriving When We Need It Most
None of us have lived through something like what we are going through today. Some ... -
FDA Grants Orphan Drug Designation to Epirium’s EB 002 for Duchenne and Becker MD
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Epirium Bio’s ... -
Capricor Hosts Call to Discuss Heart Complications of DMD, Its CAP-1002 Cell Therapy
Capricor Therapeutics will host a conference call and webcast with a cardiac expert to discuss ... -
CureDuchenne Ventures Invests in Dyne and Its DMD Therapy Program
A CureDuchenne Ventures equity investment will support Dyne Therapeutics in developing precision treatments aimed at repairing muscles in ... -
Realizing My Introversion During Self-isolation
In the days when I still attended school, I was deemed an extrovert by my ... -
MDA Partners with Invitae to Offer Free Gene Testing, Counseling for MD
Invitae and the Muscular Dystrophy Association (MDA) are partnering to expand access to free genetic ... -
Self-quarantine Has Leveled the Social Playing Field
I’ve always sought silver linings, including in this season of social distancing. Many people now ... -
MDA Hosting Online Q&A on COVID-19 Today
The Muscular Dystrophy Association (MDA) is hosting an online Q&A session today to discuss the ... -
Trial Use of Edasalonexent Not Likely to Raise COVID-19 Risk, Catabasis Reports
Clinical trial data suggest that edasalonexent — a potential Duchenne muscular dystrophy (DMD) therapy — does not ... -
Make the Most Out of Your Time – Lockdown Tips
Our Forums moderator Kevin Schaefer shared great advice for making the most out of your time ... -
The Muscular Dystrophy Community Counts on Accessibility Improvements
I feel strange not writing about the pandemic crisis in this column, but we’re already ... -
Phase 1 Results Support Losmapimod as FSHD Treatment
Final results of a Phase 1 clinical trial support the safety of losmapimod and suggest ... -
Sarepta Working to Ensure Treatment Access, Clinical Trials Continuity Amid COVID-19 Pandemic
Sarepta Therapeutics has taken steps to ensure the supply of Vyondys 53 (golodirsen) and Exondys ... -
Emflaza Supply Not Affected by COVID-19 Pandemic, PTC Therapeutics Says
The supply of Emflaza (deflazacort), a treatment for people with Duchenne muscular dystrophy (DMD), will ... -
Viltolarsen Available to U.S. DMD Patients Under Expanded Access Program
NS Pharma launched an expanded access program (EAP) in the U.S. to allow certain Duchenne muscular dystrophy ...