Updates

September 17, 2020September 17, 2020
30 Days of MD: Melissa Soriano

View this post on Instagram Day 17 of 30 Days of MD Topic: School and MD This is @thesweetlifeofmel’s story: I never thought I’d ... Read more
September 17, 2020September 17, 2020
Real-world Data Could Replace Placebo Groups in DMD Clinical Trials, Study Indicates

Real-world and natural history data could be used to supplement, or perhaps replace, the use of a placebo in future clinical trials in people ... Read more
September 16, 2020September 16, 2020
30 Days of MD: Becky Bandy

View this post on Instagram Day 16 of 30 Days of MD Topic: Love and MD This is Becky Bandy’s story: My name is ... Read more
September 15, 2020September 15, 2020
30 Days of MD: Leanne Watson

View this post on Instagram Day 15 of 30 Days of MD Topic: Changes and Challenges This is @leannes_wheel_life's story: The progressive physical effects ... Read more
September 15, 2020September 15, 2020
Sarepta Poised to Start Next Trial of SRP-9001 for Duchenne MD

Sarepta Therapeutics has completed a meeting with the Office of Tissues and Advanced Therapies (OTAT) that clarified regulatory requirements to start the company’s next ... 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

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

30 Days of MD: Leanne Watson

Sarepta Poised to Start Next Trial of SRP-9001 for Duchenne MD

30 Days of MD: Brad Miller

Overcoming the Idea of Being a Burden

30 Days of MD: Clayton Theriault

30 Days of MD: Neil Kramer

30 Days of MD: Marie De Graaf

30 Days of MD: Crystal Rondeau

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Differences in Key Proteins in DMD Muscle Cells Favor Fat Formation, Study Says

30 Days of MD: Courtney Frazer

30 Days of MD: Zee Zelinski

Santhera Acquires Global Rights to DMD Therapy Vamorolone

30 Days of MD: Briana Mills

30 Days of MD: Ainaa Farhanah Amali

30 Days of MD: Anil Jalhan

30 Days of MD: Milan Patel

September is Duchenne Action Month

30 Days of MD: Anjar Hossain

30 Days of MD: Kimberly Mullis

Same But Different Photo Contest Celebrates People With Rare Diseases

Virtual Events Mark MDA’s Muscular Dystrophy Awareness Month

30 Days of MD: Jessica Oddi

Casimersen, Exon 45 Skipping Duchenne Therapy, Under FDA Priority Review

The Battle Against Ableism Is Tiring, but I Won’t Give Up

Viltepso Commercially Available in the US for DMD Treatment

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

Why It’s Important to Be Still Amid the World’s Chaos

First Patients Enroll in Phase 1 Trial of EPM-01, Potential Becker MD Therapy

Phase 3 Trial of Pamrevlumab Enrolling DMD Patients Unable to Walk

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

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

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