Updates

November 22, 2019November 22, 2019
Method Comparing RNA Differences Between Gene Copies May Help Find Cause of Disease, Study Suggests

A new technique that compares RNA production between an individual’s two gene copies may help find genetic alterations that lead to muscular dystrophy and ... Read more
November 20, 2019November 20, 2019
Long-term Treatment with Puldysa Reduces Lung Function Decline in DMD Patients, Study Shows

Long-term treatment with Puldysa (idebenone) provides a stable reduction of lung function decline by approximately 50% in people with Duchenne muscular dystrophy (DMD), a ... Read more
November 19, 2019November 18, 2019
Improving My Digestion with Enzyme Supplements

It’s over a month since my gallbladder surgery, and I couldn’t be happier with my healing process. I previously wrote about adjusting to ... Read more
November 18, 2019November 18, 2019
NS Pharma to Provide Update on its Investigational Therapy Viltolarsen in Webinar

NS Pharma will share future plans for viltolarsen, its investigational exon-skipping therapy for Duchenne muscular dystrophy (DMD) in a webinar hosted by Parent Project Muscular ... Read more
November 18, 2019November 14, 2019
Expressing an Attitude of Thankfulness This Holiday

When I pray, I like to start by thanking God for all of the blessings he has bestowed on me. It reminds me of ... Read more

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News

PPMD Revamps Duchenne Registry with New App
November 15, 2019November 15, 2019
Parent Project Muscular Dystrophy (PPMD) announced it is updating its global registry with a ...
News

Rare Disease Film Festival Highlights Patient and Researcher Unity
November 15, 2019November 16, 2019
Rare disease-themed videos glowed on a large screen before an audience of people in ...
columns, Living, Learning, Thriving - a Column by Ralph Yaniz

Let’s Stay on the Cutting Edge of Research
November 14, 2019November 12, 2019
I can’t believe it’s been 13 years since I started exhibiting muscle loss. A ...
News

FDA Puts 2nd Clinical Hold on IGNITE DMD Trial of Gene Therapy SGT-001 Due to Patient Reaction
November 13, 2019November 13, 2019
Owing to a serious adverse event in a patient, the U.S. Food and Drug ...
News

Clinical Trial Program Testing RG6206 in DMD Boys Has Been Discontinued
November 11, 2019November 13, 2019
The clinical program evaluating the safety, tolerability, and efficacy of RG6206 in boys with ...
columns, You, Me and MD - a Column by Leah Leilani

Attack of the Bloodthirsty Mosquitoes
November 11, 2019November 8, 2019
Fall and winter have always been my favorite seasons. I eagerly await the cooler ...

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Local November 9th MDA Disease Education Events Span East, West Coast
November 9, 2019November 9, 2019
The Muscular Dystrophy Association’s (MDA) efforts to support and advocate for a wide range ...
Experimental Cell Therapy Improves Cardiac Function in Mouse Model of DMD, Study Says
November 8, 2019November 8, 2019
Treatment with a cell therapy candidate called dystrophin expressing chimeras (DECs) increased dystrophin levels ...
Investigational Therapy Losmapimod Targets Root Cause of FSHD, Early Study Suggests
November 6, 2019November 6, 2019
Goals Can Simplify Our Complicated Lives
November 4, 2019October 31, 2019
BIIB100 Improves Motor Function, Slows DMD Progression in Animal Models, Study Finds
November 4, 2019November 4, 2019
PPMD Grants Funding for Research on Cardiac Health in People with Duchenne
November 1, 2019November 1, 2019

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Prioritizing Goals in Life with MD
October 31, 2019October 29, 2019
I have spent my entire career working and volunteering in the nonprofit arena. My ...
Translarna Preserves Lung Function, Muscle Strength in DMD Boys, Real-World Data Show
October 30, 2019October 30, 2019
I Won’t Give Up My Positive Attitude for Others
October 29, 2019October 25, 2019
Vamorolone Designated Promising Innovative Medicine in UK for DMD Treatment
October 28, 2019October 28, 2019

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Novel Gene Therapy Safely Prevents Muscle Deterioration in Animal Models of DMD, Study Reports
October 25, 2019October 25, 2019
A novel one-time gene therapy that leads to the production of a dystrophin-related protein ...
Vamorolone Extension Trial Supports Muscle Gains in DMD with Fewer Side Effects, Topline Data Show
October 23, 2019October 23, 2019
Adjusting to Life Without My Gallbladder
October 22, 2019October 18, 2019

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columns, Hawk's-Eye View - a Column by Hawken Miller

Friendships Matter: You Don’t Have to Deal with Duchenne Alone
October 21, 2019October 18, 2019
I recently participated in a panel discussion at CureDuchenne 2019 Futures titled “Friends.” I ...
News

CureDuchenne Developing a Biobank for DMD Research
October 21, 2019October 21, 2019
To address the need in precision medicine for human biological specimens, CureDuchenne is creating ...
columns, Living, Learning, Thriving - a Column by Ralph Yaniz

Peeling Back the Layers of Societal Barriers for People with Disabilities
October 17, 2019October 15, 2019
We often have the radio on at home in the mornings to keep up ...
News

Positive Early Results Support Continuation of Phase 2 Trial of ATL1102 for DMD
October 16, 2019October 16, 2019
Experimental therapy ATL1102 improved upper limb strength and function in six boys with Duchenne ...
News

PPMD Grants to Promote Gene Therapy for Heart Disease and Patient Outcomes Research
October 14, 2019October 14, 2019
Parent Project Muscular Dystrophy (PPMD) has awarded two grants, one to further development of ...
News

Catabasis Joins Jain Foundation to Advance Edasalonexent Development for Dysferlinopathy
October 11, 2019October 11, 2019
Catabasis Pharmaceuticals and the Jain Foundation have started a preclinical research collaboration to study ...
News

Dyne Therapeutics Supporting FSHD Natural History Study Underway in US, Europe
October 10, 2019October 18, 2019
Dyne Therapeutics announced that it has joined in supporting an ongoing global and observational natural ...
News

Gene Therapy SRP-9003 Showing ‘Very Encouraging’ Results at 9 Months in Limb Girdle MD, Sarepta Reports
October 9, 2019October 9, 2019
New nine-month data from a Phase 1/2 trial show that a single administration of ...
columns, Hawk's-Eye View - a Column by Hawken Miller

The Right Job Can Help You Find Fulfillment and Purpose
October 7, 2019October 3, 2019
I’ve just wrapped up my internship with The Washington Post. My experience has made ...

May 21, 2018June 3, 2019

FDA Grants Rare Pediatric Disease Status to Myonexus’ MYO-101 for LGMD 2E

News

The U.S. Food and Drug Administration recently granted rare pediatric disease designation to Myonexus Therapeutics’ experimental gene therapy MYO-101 for the treatment of limb girdle muscular dystrophy (LGMD) type 2E, or LGMD2E. The announcement was made by Myonexus and Nationwide ... Read more

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