Updates

January 14, 2021January 14, 2021
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, in treating Duchenne muscular dystrophy (DMD). “The initiation ... Read more
January 12, 2021January 7, 2021
I Make Sure My Support System Is Strong

When I’m deep in thought, reflecting on my life, I shudder to think who and where I’d be without the support of my family ... Read more
January 12, 2021January 12, 2021
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 dystrophin in boys with Duchenne muscular dystrophy (DMD) ... Read more
January 11, 2021January 11, 2021
NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual ... Read more
January 11, 2021January 7, 2021
Why Freelancing Works for My Life With Duchenne

Since my internship at The Washington Post, I’ve done freelance work at outlets ranging from The Forward, a Jewish-focused website, to Dot Esports, and ... Read more

Subscribe to our mailing list

Get regular updates delivered to your email box.

Thanks! Please check your email for a confirmation email.

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

Updates

Dosing Begins in Phase 3 Trial of Pfizer’s Gene Therapy for Duchenne

I Make Sure My Support System Is Strong

DMD Boys Experience Biological, Functional Gains in SRP-9001 Study

NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

Why Freelancing Works for My Life With Duchenne

Pivotal Trial Begins for AMO-02 in Steinert Disease

Top 10 Muscular Dystrophy Stories of 2020

Edgewise Will Use $95M Funding to Advance EDG-5506 for BMD, DMD

It’s Time to Spread Some Holiday Cheer

Genethon Cleared to Launch French Trial Testing GNT0004 Gene Therapy for DMD

Lower-dose SRP-5051 Potentially More Effective Than Exondys 51, Phase 2 Trial Finds

Subscribe to our mailing list

Translarna Approved in Russia for DMD Patients 2 and Older

Lilly, Precision to Develop Gene Editing Therapies for DMD, Other Disorders

Give Yourself Some Peace of Mind This Holiday Season

How to Avoid a Fall

MDA Wants Neuromuscular Disease Patients to Have Early Access to COVID-19 Vaccine

ReveraGen Receives $3.3M NIH Grant to Advance Vamorolone in DMD

NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting

Sarepta Talks Progress, Plans for New Trials of DMD Gene Therapy SRP-9001

Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

Looking at Holiday Self-isolation in a Positive Light

DM1 Patients Asked to Join Natural History Study Aiming for Better Clinical Trials

Expressing Our Negative Emotions to Find Positive Ones

Phase 1 Trial of Oral EDG-5506 for Becker MD Enrolling at Texas Site

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

Q&A: Meet the Muscular Dystrophy Association’s New CEO and President

MDA Launches Tool to Make Clinical Data More Accessible to Researchers

Saying Goodbye to Eye Strain, Thanks to Blue-light Glasses

AMO-02 for Congenital DM1, Entering Pivotal Trial, Gets Rare Pediatric Disease Status

How I’m Getting Out of My 2020 Funk

Ultragenyx and Solid Biosciences Partner on DMD Gene Therapy

NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits

PPMD Seeking Adults with Duchenne or Becker MD to Join Advisory Committee

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