Updates

November 26, 2020November 26, 2020
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 anxiety in people with rare diseases, negatively affecting their ... Read more
November 24, 2020November 20, 2020
Looking at Holiday Self-isolation in a Positive Light

The holidays are notorious for being one of the most hectic times of the year. People jump at the opportunity to score the best ... Read more
November 24, 2020November 24, 2020
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 (DM1) to participate in a large natural history ... Read more
November 23, 2020November 19, 2020
Expressing Our Negative Emotions to Find Positive Ones

I always hope to impart a positive message to my readers and exude joy in life whenever possible, but I also know it’s OK ... Read more
November 19, 2020November 19, 2020
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 and tolerability of EDG-5506, its investigational oral treatment for ... Read more

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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

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

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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

Phase 3 Trial Failure Prompts Catabasis to Stop Edasalonexent Development

Long-term Corticosteroid Use Delays Lung and Heart Decline in DMD Boys, Study Finds

UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner

Rapper Wheelz Hosts Oct. 24 ‘Ryan’s Day’ Virtual Fundraiser for DMD

The 4 Categories of Tiredness

MDA Telethon Returning, Going Virtual With Host Kevin Hart

Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

What I Learned About Life From My Dog

Edasalonexent Helps to Preserve Heart and Bone Health, New Data Show

Phase 3 Trial of Puldysa Stopped for Lack of Efficacy in Duchenne Patients

Pfizer’s Gene Therapy for Duchenne Placed on Fast Track by FDA

Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos

FDA Lifts Clinical Hold on IGNITE DMD Gene Therapy Trial

Living for Myself While Hoping for the Right Person

Removing Barriers to Employment for People With Disabilities

Durable Safety and Motor Gains Evident in Trial of SRP-9001, DMD Gene Therapy

30 Days of MD: Miroslava Zerova

Givinostat Wins Rare Pediatric Disease Designation; Phase 3 DMD Trial Fully Enrolled

30 Days of MD: Alireza Soli

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