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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) ...
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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 ...