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US Patient Groups Give Thumbs-Up to Rule Against Surprise Billing
The National Organization for Rare Disorders (NORD) is applauding the Biden administration for announcing a ...
More from Huntington’s Disease News
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Wearing Down of Protective System in Brain May Underlie Huntington’s
Progression in Huntington’s disease may be due more to a gradual loss of the system that maintains ... -
A Road Trip I Would Only Take for My Mom
I’ve found that having a parent with Huntington’s disease makes you value every moment with ... -
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 ... -
Cleveland Hope Walk & 5K Run Set for Aug. 15, Benefits HDSA
The Cleveland Team Hope Walk & 5K Run is on tap for Aug. 15 to ... -
Why I Choose to Be Like Water
“Parting is such sweet sorrow,” Shakespeare once wrote. And don’t I know it. Recently, the ... -
Nominations Open for 2022 Eurordis Black Pearl Awards
Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. ... -
Brain-Derived Neurotrophic Factor Levels ‘Unlikely’ as Valid Biomarker
Brain-derived neurotrophic factor (BDNF) may not be the biomarker for Huntington’s disease that it was previously thought ... -
The Squeaky Wheel Gets the Grease
Dear people working to find a cure for Huntington’s disease (HD), I imagine you all ... -
My Experience Participating in an Observational Study
One afternoon in May, I got a call from a number I didn’t recognize, and ... -
Past Alcohol Abuse Linked to Motor, Psychiatric Problems in Patients
A history of high alcohol intake seems to be linked to increased motor impairment and ... -
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 ... -
Preclinical Research Supports Pridopidine’s Therapeutic Potential
Treatment with pridopidine rescues the function of mitochondria — the cell’s powerhouses — boosting energy production, ... -
Good Decision-making Is an Important Trait for Caregivers
My wife, Jill, and I recently bought a new car. While this might not seem ... -
EveryLife Introduces First of Kind ‘Roadmap’ to ICD Codes
To help patient advocacy leaders and their partners better understand how global health statistics codes ... -
Phase 1/2 Trial of AMT-130 Gene Therapy Enrolling High-dose Group
The first two patients have been enrolled in the high-dose group of a U.S. clinical trial ... -
Look! A Squirrel!
My wife, Jill, has diagnosed me with attention deficit hyperactivity disorder (ADHD). (Look! A squirrel!) ... -
Why I Dislike Using the Word ‘Hope’ When Discussing Huntington’s Disease
I’ve been a part of the Huntington’s disease (HD) community since my mom was diagnosed ... -
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 ... -
Galyan Bio Plans to Start Clinical Testing of Oral Therapy in 2022
Galyan Bio plans to initiate a clinical trial of a small molecules candidate that aims to slow the ... -
How Huntington’s Is Like a Cyberattack
In early May, cybercriminals known as DarkSide launched a devastating ransomware attack on Colonial Pipeline, ... -
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 ... -
Levels of KP Metabolites in Blood, CSF Unlikely as Disease Biomarkers
Metabolites seen as potential biomarkers of Huntington’s disease — called kynurenine pathway metabolites — showed little ... -
Since Her Diagnosis, My Wife Has Benefited From an Antidepressant
The school year recently ended, which may be good for students and teachers, but not ... -
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 ... -
While Buying a Condo, I Was Reminded of the Importance of Teamwork
Last weekend, I had a bunch of newly vaccinated friends over to help me paint ... -
Board OKs AMT-130 Gene Therapy Trial to Enroll for High-dose Group
A Phase 1/2 trial testing the safety and efficacy of AMT-130, uniQure’s experimental gene therapy for Huntington’s ... -
PROOF-HD Phase 3 Pridopidine Trial Reaches 25% Enrollment
Prilenia Therapeutics has enrolled more than 120 patients in PROOF-HD, a Phase 3 clinical trial ... -
Playing Games Is Serious for My Wife
My wife, Jill, and her phone are like Siamese twins — attached all the time. ... -
Getting Involved in Community Events Is Hard for Me
I’ve written before that I was unsure if I was ready to get involved in ... -
When Parent and Child Disagree on Genetic Testing for Huntington’s Disease
Getting the Huntington’s disease genetic test is a major life decision that weighs heavily on ... -
1st Group Enrolled in Trial of uniQure’s AMT-130 Gene Therapy
uniQure has completed patient enrollment for the first group participating in a Phase 1/2 trial ... -
Why Empathy Is an Important Part of Huntington’s Disease Awareness Month
I recently gave a presentation at an event, during which I explained that my wife ... -
Nonprofit Pushes for US Bill to End Wait for Patient Benefits
U.S. lawmakers have again introduced legislation that would speed up the process for Huntington’s disease ... -
Time Flies When You Want It to Slow Down
This week, my wife, Jill, bought meals from HelloFresh, a company that sells kits with ... -
Decision to Stop Dosing in Tominersen Trial ‘Pretty Hard to Take’
Sarah Lynn Mays learned she wouldn’t be returning to Northwestern University for her scheduled intrathecal injection ... -
GENERATION HD1 Trial Fails to Show Benefits of Tominersen
Treatment with tominersen failed to show evidence of clinical benefit in adults with Huntington’s disease during ... -
‘Let’s Talk About’ Huntington’s Disease Awareness in May
Activities are underway worldwide to mark Huntington’s Disease Awareness Month, set aside each year to ... -
The Importance of Preserving Our Words
I recently received an email from Kelli Yoder, the communications manager at Adira Foundation, an ... -
How the HEATED Project Can Benefit the Huntington’s Community
As I wrote in a previous column titled, “An Abundance of Huntington’s Disease Info Awaits ... -
FDA Gives Go-ahead for Phase 1/2 Trial Testing VY-HTT01 Gene Therapy
The U.S. Food and Drug Administration (FDA) has granted Voyager Therapeutics permission to start a clinical ... -
Collecting Memories to One Day Look Back on ‘The Way We Were’
Buried in my brain is a Proustian memory. It occurred during my elementary school graduation ... -
Writing Down My Fears About Huntington’s Makes Them Worse
I learned about my mother’s Huntington’s diagnosis and my own risk during my senior year ... -
Quick Approval Given COVID Vaccines Raise Concerns, Rare Disease Patients Say
More than half of the people with rare diseases and their caregivers, asked in a ... -
Protein With ‘Switch’ Sensitive to Clumping May Aid in Treating Disease
A chaperone protein called DnaJB8 has an internal molecular “switch” that likely regulates how cells ... -
Don’t Believe Everything You Read (Except This Column)
One of the characteristics of a good journalist is being able to digest a lot ... -
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 ... -
Consortium Will Promote Stem Cell Therapies
A new international consortium, Stem Cells for Huntington’s Disease (SC4HD), has been formed to help ... -
2 Valuable Caregiving Lessons From My Mother-in-Law
Over Easter weekend, my mother-in-law, Edwina, visited. My wife, Jill, and I are always happy ... -
Huntington’s Has Strengthened My Relationship With My Mom
Having a parent with Huntington’s disease influences my life in many ways, most of which ... -
Anima Biotech, Takeda Team Up to Develop Small Molecule Treatments
Anima Biotech has partnered with Takeda Pharmaceuticals to develop small molecules that control mRNA translation ... -
Believing in Miracles Helps Me Overcome Despair
“Do you believe in miracles?” Sports announcer Al Michaels uttered that sentence on national television ... -
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 ... -
Dosing Stopped in Phase 3 Trial of Tominersen for Huntington’s, Roche Says
Roche has discontinued dosing in the Phase 3 GENERATION HD1 trial of its investigational therapy tominersen in ... -
Focusing Your Energy on Others Brings Inevitable Joy
My wife, Jill, and I do not dwell on her Huntington’s disease (HD). We know ... -
Mutant Huntingtin Slows Cells’ Protein-Building Machinery, Study Finds
Stalled protein-building may underlie Huntington’s disease, according to data from a recent study. The findings ... -
The Power of Kindness Can Change the World for Caregivers
One of the most inspirational definitions of kindness I’ve ever read comes from Father Lawrence ... -
I Keep Moving by Storing Little Boxes in My Head
I like to keep things organized. Everything should have a place, even if it’s on ... -
A Former Huntington’s Caregiver Shares Her Best Caregiving Tips
I like learning, especially from smart people. My wife, Jill, is one of the smartest ... -
Women with Huntington’s Experience Worse Symptoms Than Men, Global Study Suggests
Women with Huntington’s disease have worse motor, cognitive, and depressive symptoms than men with the ... -
An Important Lesson I Learned From Waiting at the Doctor’s Office
I have previously discussed in this space my wife Jill’s dislike of visiting the doctor. ... -
Project Seeks Contributors to Depict Life With Huntington’s
An alliance of Huntington’s disease (HD) organizations in the U.K. is calling for patient contributions ... -
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 ... -
We All Deserve Quality Care: A Deep Dive Into ‘The Soul of Care’
I love to read. I developed my affinity for books as a young child, when ... -
Wearable Devices May Aid Diagnosis, But Need Improvement
Wearable devices show considerable promise in helping diagnose people with Huntington’s disease, but the studies ... -
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 ... -
Here’s How You Can Get Involved in Non-clinical Huntington’s Research
In a previous column, I talked about the many questions one might have when signing ... -
HDSA Expands Centers of Excellence Program to 54 Sites
With some recent additions, the Huntington’s Disease Society of America (HDSA) has expanded its Centers ... -
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 ... -
My Greatest Dream Is to Eliminate Rare Disease Day
One of U.S. President John F. Kennedy’s dreams was to send men to the moon ... -
Eurodis Survey: Healthcare Experience Worse for Rare Disease Patients
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, ... -
Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, ... -
Let’s Dance: The Pope and HD Families Meet in a Special Film
“Dancing at the Vatican” is a 2019 documentary film created and narrated by Charles Sabine, ... -
Safety Seen in AMT-130 Gene Therapy Trial, Six More Patients to Enroll
No significant safety concerns were found in the first four patients participating in a Phase ... -
An Abundance of Huntington’s Info Awaits at Upcoming Virtual Conferences
Every day, people around the world learn more about Huntington’s disease (HD). We know so ... -
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 ... -
A Valentine’s Day Love Letter to My Dear Wife
In honor of Valentine’s Day last year, I wrote my wife a love letter. I’ve ... -
UK Guidelines Look to Better Mental Health of Huntington’s and Other Patients
The British Psychological Society has released evidence-based recommendations for health professionals managing the psychological health of people with ... -
Adira Foundation Arises From Life-altering Accident to Help Those With Chronic Ills
Greg Smiley’s world changed abruptly in 2013. He was racing down a mountain road on ... -
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 ... -
Here Are the Results From My 21-day Negativity Detox
Late last year, my wife, Jill, and I challenged ourselves to a 21-day negativity detox. ... -
More MOR1 May Contribute to Psychiatric, Motor Symptoms
Higher-than-normal levels of mu-opioid receptor 1 (MOR1) in the striatum — a brain region particularly ... -
Got Questions About Joining a Huntington’s Clinical Trial?
There is a robust pipeline of potential therapies for Huntington’s disease (HD), which I’ve discussed ... -
My Project to Create a Healthy Emotional Outlet for Caregivers
One positive thing about having a Cuban mother is eating Cuban food. My mom is ... -
PET Scan Used to Distinguish Juvenile HD From Parkinson’s in Case Study
A teenage boy with a history of parkinsonism was diagnosed with juvenile-onset Huntington’s disease (HD) using high-sensitivity ... -
NORD’s Caregiver Respite Program Continues Through Pandemic
Caring for a loved one with a rare disease, especially during these uncertain times, demands ... -
Losing My Sense of Taste Taught Me a Lesson in Caregiving
Last November, I joined a club. I share this news with the same spirit that ... -
Study Links Cognitive Impairment to Reduced Gray Matter Volume
Based on MRI scans, Huntington’s disease patients with major cognitive impairments have reduced gray matter ... -
UK Rare Diseases Framework Opens to Improve Diagnosis, Access to Care
U.K. Rare Diseases Framework, a government-led approach to raising national awareness of rare disorders, accelerating ... -
No Matter Your Hardships, Today Is a Great Day to Be Alive
I’ve lost two moms in my lifetime. My biological mom, Debbie, who was with me ... -
Huntexil Analysis Shows Slowed Decline in Total Functional Capacity
Treatment with Huntexil (pridopidine) can slow the decline in total functional capacity in people with ... -
As Caregivers, We Can Choose a Positive Mindset
It’s all about the mindset. I suggest caregivers work on developing a positive mindset. It ... -
Focusing on What Is Good and Beautiful This Year
Hi, 2021. How are you? You have much to live up to. Last year, a ... -
‘Dancing at the Vatican’ Spotlights Families’ Struggles, Joy at Meeting Pope
The Huntington’s Disease Association of Ireland (HDAI) is spreading the word about a new documentary ... -
Operation Warp Speed Should Inspire a Similar Effort for Rare Diseases
Whoever named Operation Warp Speed, the U.S. government’s project to produce and develop COVID-19 vaccines ... -
Natural History Study of Huntington’s Fully Enrolled, Triplet Says
Triplet Therapeutics announced it has completed patient enrollment in SHIELD HD, a natural history study ... -
Getting Involved in the Huntington’s Community Brings Great Rewards
I have only known one way to live in a family affected by a rare ... -
Single CDNF Protein Injection Shown to Protect Nerve Cells, Improve Motor Skills in Rats
A single injection of the protein CDNF — cerebral dopamine neurotrophic factor — into the brains ... -
The Turn-Negatives-Into-Positives Challenge
Thick rubber bands seem unbreakable. It takes effort to pull each end. In contrast, breaking ... -
Time Is the Best Gift to Share With Others
So … December is finally here. It’s the last month of the longest year ever, ... -
1st Patient Dosed in Phase 2a Trial of Annexon’s Antibody Therapy for Huntington’s
The first participant was dosed in a Phase 2a clinical trial testing Annexon’s investigational therapy ANX005 ... -
Love Is the Mission
I usually write about what life is like as a caregiver, along with my observations ... -
Late-onset Huntington’s Has Fewer Motor Defects, Similar Cognitive Progression, Study Shows
Patients with early-onset Huntington’s disease (HD) experience more muscle and eye movement abnormalities, and more ... -
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 ... -
Would You Want to Know If You Have Huntington’s?
Editor’s note: This column briefly mentions the topic of suicide. Please find resources for help ... -
I’m Determined to Focus on Gratitude
Two years ago, my wife, Jill, took a genetic test and discovered she has Huntington’s ... -
Phase 1 Trial Will Test Oral PTC518 Candidate Therapy; First Results Due Early 2021
A Phase 1 trial will evaluate PTC Therapeutic’s PTC518, its investigational therapy for Huntington’s disease, ... -
The Value of Consulting a Genetic Counselor During Testing
As I briefly touched on in my previous column, “Opportunities for a Better Genetic Testing ... -
The Definition of a Caregiver
At some point in the history of mankind, someone said, “Let’s raise awareness about [topic ... -
PDYN-derived Peptides Identified as Possible, Much-needed HD Biomarkers
Levels of proteins derived from prodynorphin (PDYN) — ultimately used during cell-to-cell signaling in the ... -
Paying Attention to My Wife’s Silent Suffering
The events of the past week have reminded me just how tough my wife, Jill, ... -
First Patients Enrolled in PROOF-HD Phase 3 Trial Testing Pridopidine
The first U.S. patients have been enrolled in PROOF-HD, a Phase 3 clinical trial investigating ... -
Fasting as a Means of Solidarity
When my sister died in a tragic accident a year ago, grief caused me to ... -
1st Wexler Prize Given Scientist Studying Astrocytes Role in Huntington’s
The Hereditary Disease Foundation‘s (HDF) has announced the first winner of its Nancy S. Wexler Young Investigator ... -
Opportunities for a Better Genetic Testing Process
I’ve always tried to stay alert to the needs of the Huntington’s disease community by ... -
UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner
A photo of a bespectacled young boy, his red baseball cap slightly askew as he ... -
Making a Difference in the Upcoming Elections
When I write this column every week, I try to only share the things that ... -
Branaplam Receives Orphan Drug Designation for Huntington’s; Trial Planned for 2021
The U.S. Food and Drug Administration (FDA) has given orphan drug designation to the oral ... -
Should I Get a Huntington’s Tattoo?
When I was a sophomore in college, I debated going blond. It was January, and ... -
FDA Puts Voyager’s Gene Therapy Candidate VY-HTT01 on Clinical Hold
The U.S. Food and Drug Administration (FDA) is putting on hold Voyager Therapeutics’ request to test VY-HTT01, its investigational ... -
It’s Not About You … It’s About Me
I have decided to get my wife a T-shirt that reads, “It’s not you, it’s ... -
Mental Preparedness Is Crucial Ahead of Genetic Testing
As I mentioned in my debut column of “Negative to Positives” a couple weeks ago, ... -
Safety Board Recommends AMT-130 Trial Continue Enrolling, Treating Patients
No significant safety concerns associated with AMT-130, uniQure’s experimental gene therapy for Huntington’s disease, were identified ... -
The Protective Mindset of a Caregiver
I’m protective, a trait typical of most men. For example, I will drive for 18 ... -
Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos
Same But Different, a nonprofit U.K. group that uses art for social change, is inviting ... -
Show, Don’t Tell: The Little Details That Count in Caregiving
When I first understood that I was going to be a writer, I did what ... -
Top-line Data Show Potential Cognitive Benefit of Pepinemab in Early Huntington’s Patients
Vaccinex’s investigational antibody pepinemab showed good tolerability in a Phase 2 clinical trial of people ... -
‘The Good Place’ Reminds Me of the Importance of a Moment
On Saturday, the last season of the Netflix series “The Good Place” began streaming. To ... -
Welcome to ‘Negative to Positives,’ A New Huntington’s Disease Column
Like many of you reading, I am from a Huntington’s disease (HD) family. I learned ... -
Life Is Short, So Live It Well
The anniversary of my sister’s death is coming up in about a month. Rose died ... -
Phase 3 Trial of Oral Pridopidine in Early Stage Patients Opening Soon
Prilenia Therapeutics, in partnership with the Huntington Study Group (HSG), has launched the Phase 3 ... -
Learning to Expect the Unexpected
My wife, Jill, and I have not been to the local movie theater in months. ... -
New book, ‘Harry Needs a Hug,’ Helps Children Who Have Huntington’s in Their Family
A new book is available to help children cope when a family member has Huntington’s ... -
Finding a Therapist to Talk to About Huntington’s Can Be Hard
This week, I have my first therapy appointment in a while. I hope therapy will ... -
Writing Keeps This Caregiver Sane
I stumbled into my future profession when I took a creative writing class as a ... -
Huntington’s in Youth Closely Tied to Problems With School and Social Life
Behavioral changes due to early onset Huntington’s disease and problems with school, and social and family ... -
Learn How to Party On, Dude
One of summer’s guilty pleasures is watching mindless movies. On a recent weekend, Jill and ... -
TBK1 Enzyme Seen to Clear Mutant Huntingtin Protein in Disease Models
An enzyme called TANK‐binding kinase 1 (TBK1) can lower levels of the mutant huntingtin protein ... -
An Ant Invasion Reminds Me How Important Having My Rock Is
About a week ago, my boyfriend and I treated ourselves to Qdoba for lunch. I ... -
As Caregivers, We Can Choose to Be Empathetic and Patient
My mom is dying. She’s 90, has dementia, can’t walk, yells angrily at times, and ... -
Huntington’s Alters Brain Development Before Birth, Study Shows
Huntington’s disease alters nerve cell development in the cortex — a brain region largely affected ... -
Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input
Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed ... -
Gut Microbiome Seen to Differ in People With Huntington’s or at Disease Risk
Microbes in the gut of people with Huntington’s or likely to develop this disease appear to ... -
Telemedicine Makes Life Easier for Patients
Last year, I wrote about the possibility of telemedicine visits for Huntington’s patients with the ... -
Striving to Be as Carefree as a Kitten
I have been working from home since March and likely will continue to do so ... -
Living Outside the Box
If you could go anywhere on vacation, would you take your time planning the perfect ... -
Antidopaminergic Meds Ease Chorea But May Worsen Cognition, Study Finds
Antidopaminergic medications are associated with lesser chorea and irritability in people with Huntington’s disease, but they may worsen ... -
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 ... -
New Streaming Channel Showcases Rare Disease Films
When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated ... -
Gene Transfer Method of EPO Improves Cognition in Mouse Study
Administration of the hormone erythropoietin (EPO) using a gene transfer method improved spatial cognition, stimulated ... -
The Enduring Positivity of Huntington’s Patients and Families
Ever since my wife, Jill, and our daughter were tested and diagnosed as gene positive ... -
I’ve Learned to Focus on Living in the Moment
I spent about two months weighing the pros and cons of getting bangs again. On ... -
Vico Therapeutics Raises $31M to Fund Advances in Central Nervous System Treatments
Vico Therapeutics has raised $31 million in Series A financing to continue pursuing the development ... -
Respecting Religious Beliefs Can Offer Peace in Families
What makes a great marriage? That age-old question has been the subject of many books ... -
Lessons Learned from Watching ‘Scooby-Doo’
Did you ever watch cartoons when you were young? One of my favorites as a ... -
Study Finds Evidence of Brain Damage in Huntington’s Decades Before Clinical Onset
People who will develop Huntington’s disease have subtle biological evidence of brain damage decades before ... -
Welcome to Empty-Nest World
If you think adulting is hard, wait until you are empty nesting. My wife, Jill, ... -
Adequate Care Lacking for Many With Huntington’s in India, Study Finds
Among people with Huntington’s disease living in India, psychiatric problems are common but routine clinical ... -
My Support Network Keeps Me Sane
During college, I always joked with my friends that I would drop out of school ... -
The Importance of Food, Glorious Food
The last few weeks have been somewhat trying for Jill. She has been plagued with ... -
Natural History Study of Huntington’s Starts Enrolling Participants
The first patients have been enrolled in a clinical study of the natural history of ... -
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 ... -
AFFiRiS’ New Antibody May Help Halt HD Progression, Early Data Suggests
AFFiRiS’ C6-17, a candidate monoclonal antibody for the treatment of Huntington’s disease, is able to target and ... -
Being a Caregiver Means Being Involved
One of my recent columns was about the Huntington’s Disease Society of America’s annual convention. ... -
The Simple Joy of Taking a Walk
We live in Illinois, where the weather can be unpredictable. Some days we have perfect ... -
AMT-130, uniQure’s Gene Therapy, Given to First Patient Group in Trial
A Phase 1/2 clinical trial evaluating AMT-130 as a potential gene therapy for Huntington’s disease has “treated” its first ... -
It’s Not About You, Caregivers
I am messy. I’ve always been messy. My desk at work looks like an explosion ... -
German Researcher Promotes Non-drug Alternatives for Treating Huntington’s
At least three major pharmaceutical companies are looking into potential gene therapies for Huntington’s disease ... -
Cooking Reminds Me of Cherished Time with My Mother
My grandmother and my mom taught me how to cook. Growing up, I would make ... -
The Day I Summoned the Courage to Update My LinkedIn Profile
We were settling down in the living room to relax after putting our daughter to ... -
Overcoming Denial in the Face of Huntington’s Disease
The Huntington’s Disease Society of America held its annual conference in early June. Like many ... -
Austedo to Manage Chorea Safe at Higher Doses, Study Finds
Austedo (deutetrabenazine) can be safely taken at higher than the maximum dose now approved to ... -
To Join or Not to Join, That Is the Question
To this day, I have only talked to two younger people with Huntington’s who weren’t ... -
Coalition Will Address Racial Disparities in Rare Disease Communities
The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on ... -
The Real World Is Precious
On May 21, 1992, MTV aired the first episode of “The Real World.” The introduction ... -
‘Hero’ Proteins Protect Other Proteins from Stress, Research Finds
A novel class of proteins, dubbed heat-resistant obscure (Hero) proteins, can protect other proteins from ... -
Pepinemab Clinical Trial Ongoing; Top-line Results Coming Later this Year
Vaccinex’s Phase 2 SIGNAL study, evaluating the investigational antibody pepinemab (VX15/2503) in Huntington’s disease patients ... -
A Caregiver’s Biggest Lesson
I have pink eye. Again. And I’m extremely happy I got it. I’ll get to ... -
COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say
While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for ... -
Feeling Helpless Vs. Being Helpful
If you’ve ever experienced an allergic reaction to something, you probably understand why the root ... -
China Approves Austedo for Huntington’s-related Chorea
Teva Pharmaceuticals’ Austedo (deutetrabenazine tablets) has been approved by China’s National Medical Products Administration (NMPA) for treating ... -
Pulled Between Two Lives
Working remotely, while slightly draining, has had its benefits. Being able to sleep in until ... -
Music Transports Me Back to Those Dark Diagnosis Days
My husband and I were driving along a country road with no destination in mind. ... -
The Inexhaustible Reality of Love
Reality bites. That was the name of a 1994 romantic comedy movie starring Winona Ryder ... -
Government Agencies Approve MMJ’s Import of Marijuana for Research
Multiple government agencies have cooperated to allow MMJ International Holdings to import cannabis extracts to ... -
Honoring a Huntington’s Family Member Dedicated to Finding a Cure
May is Huntington’s Disease Awareness Month. While I was starting to write this week’s column, ... -
Huntington’s Disease Awareness Month Goes Virtual in May Due to Pandemic
Because of the COVID-19 pandemic, Huntington’s Disease Awareness Month organizers have come up with creative ways ... -
Rare Diseases Clinical Research Network Opens Online Survey on COVID-19
The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand ... -
Practicing Self-care and Letting Go of the Guilt
All of my friends know about my love of wine. The first summer after I ... -
Hugging Is One of the Things I Miss the Most
The first time I met my wife, Jill, she told me she wasn’t comfortable with ... -
AskBio Acquires BrainVectis and Potential Gene Therapy for Huntington’s
AskBio announced that it has acquired BrainVectis, a company developing gene therapies for Huntington’s disease and other neurodegenerative conditions. The ... -
Enrollment Complete for GENERATION HD1 Trial Testing Tominersen for Huntington’s
Roche has completed patient enrollment for its Phase 3 GENERATION HD1 clinical trial evaluating the potential of ... -
Forced Family Time
My wife, Jill, and I live in Illinois, where the governor has declared a stay-at-home ... -
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 ... -
My Genetic Status Complicated Having a Baby
As I walked down the street to get my afternoon coffee one day, it appeared ... -
The Realities of Rare Diseases
Social distancing seems to be the trend these days. Several reality shows illustrate what I ... -
Vaccinex’s Pepinemab Halts Brain Shrinkage, Eases Symptoms in Early Huntington’s Patients, Results Shows
Vaccinex’s experimental therapy pepinemab (VX15/2503) improves brain metabolism, halts its atrophy, and eases motor and ... -
New Perspectives from a Trip to the ER
This past Monday night, my boyfriend and I cooked an amazing meatball dish with garlic ... -
Small Molecule Seen to Reverse Huntington’s Repeat CAG Mutation
A DNA-binding compound can reverse the repeat expansion mutation that is associated with Huntington’s disease, ... -
We Are Two People with One Heart
My home has been super quiet for the past several days, a silence that will ... -
Believing in the Impossible
When she was little, my daughter, Alexus, visited a 7-Eleven store with my wife, Jill. ... -
Factor-H Reaches Out to Huntington’s Neglected Families of South America
Huntington’s disease affects an average of 1 in 10,000 people worldwide. But in small, remote ... -
A 10-minute Walk, and a Purr That Changed My Life
Thursday, Oct. 17, 2019, started off like most weekdays for me. Working in consulting, there ... -
The Importance of Having ‘Emotional Vitamins’
I can’t stand dogs. They bark a lot. They smell. They require a lot of ... -
AMT-130 Gene Therapy Trial Postpones Treatments Amid COVID-19 Pandemic
Treatment in a U.S.-based Phase 1/2 clinical trial evaluating uniQure‘s gene therapy AMT-130 in people ... -
Pain Management Often Neglected in Huntington’s, Researchers Say
The uncontrolled jerking and writhing movements associated with Huntington’s disease are well known, along with ... -
Saying ‘Yes, Yes!’ to the Power of Words
I wasn’t expecting to be brought to tears when I heard motivational speaker Lisa Nichols ... -
Celebrating a Year of Being Part of a Courageous Community
This week marks a year since I started writing my column, “A Family Tradition.” Having ... -
‘Our Odyssey’ Nonprofit Connects Young Adults Who Have Rare Diseases
As a high-school student in the suburbs of Boston, Seth Rotberg watched his mother’s mental ... -
Coronavirus Has Affected Everyone’s Lives
For many people I know, including me, COVID-19 started out as a joke. It shares ...