Updates

October 20, 2020October 16, 2020
The 4 Categories of Tiredness

Those of us with invisible chronic illnesses master the art of mindfulness for our bodies. Since our symptoms show little signs of existence, we ... Read more
October 20, 2020October 20, 2020
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 Hart hosting the virtual event. The two-hour MDA Kevin ... Read more
October 19, 2020October 19, 2020
Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its ... Read more
October 19, 2020October 15, 2020
What I Learned About Life From My Dog

On Sept. 29, part of my life was lost. My dog, Gus, who had been a staple of the Miller family household for 13 ... Read more
October 15, 2020October 15, 2020
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 heart disease and help to preserve bone health, ... 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

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

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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

Efforts Continue Beyond Awareness Month to Expand Newborn Screening Panels in US

30 Days of MD: Colette Wheeler

30 Days of MD: Jason Revill

30 Days of MD: Allie Schmidt

30 Days of MD: Noelle Grace

30 Days of MD: Kae Tran

Vamorolone Continues to Improve Motor Function in DMD Boys, Phase 2 Trials Show

30 Days of MD: Joe Logue

30 Days of MD: Vincent Kelly

Muscular Dystrophy and Other Clinical Trials Adapting to Life With COVID-19

30 Days of MD: Cassie Brode

The Value of Having Friends With the Same Disease

30 Days of MD: Tali Maron

30 Days of MD: Jonathan “2J” Groenewald

30 Days of MD: Jenna Milidan

30 Days of MD: Melissa Soriano

Real-world Data Could Replace Placebo Groups in DMD Clinical Trials, Study Indicates

30 Days of MD: Becky Bandy

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

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