duchenne muscular dystrophy (DMD)

Sarepta Advances Precision Genetic Therapies Toward Full FDA Approval: AMONDYS 45 and VYONDYS 53 Seek Traditional Approval Status

Post author:Bree Clare
Post published:April 20, 2026
Post category:Duchenne Muscular Dystrophy

Sarepta Therapeutics has announced a significant regulatory milestone in its efforts to secure permanent FDA approval for two breakthrough Duchenne muscular dystrophy (DMD) treatments. As reported by BusinesWire.com, the company…

Long-Term Study Shows Givinostat’s Promise for Duchenne Muscular Dystrophy Treatment

Post author:Bree Clare
Post published:August 30, 2025
Post category:Duchenne Muscular Dystrophy

ITF Therapeutics has announced the publication of encouraging long-term data supporting the efficacy and safety of givinostat as a treatment for Duchenne muscular dystrophy (DMD), according to a recent article…

Young Advocate: Ben’s Mission to Champion Kids’ Health in Washington, DC

Post author:Bree Clare
Post published:June 27, 2025
Post category:Duchenne Muscular Dystrophy

Not everyone who stands up for children’s health in Washington, DC, wears a suit or carries a briefcase. Sometimes, the most powerful advocates are the ones who have experienced the…

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A Parent’s Take on Marriage While Having a Child with Duchenne Muscular Dystrophy

Post author:Alyssa Stevens
Post published:February 14, 2022
Post category:Duchenne Muscular Dystrophy Muscular Dystrophy

Betty Vertin recently wrote an article discussing her experience as a parent of a child with Duchenne muscular dystrophy. She shares the lessons she has learned in order to…

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First Patient Dosed in Phase 3 Study of Experimental Idiopathic Pulmonary Fibrosis Drug

Post author:Scott Carlson
Post published:July 29, 2019
Post category:Duchenne Muscular Dystrophy Idiopathic Pulmonary Fibrosis

According to a press release from the San Francisco-based FibroGen, Inc., the first patient has been dosed in the Company's phase 3 clinical study of the experimental drug pamrevlumab for…

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Family Touched By Muscular Dystrophy Sets Up GoFundMe

Post author:Scott Carlson
Post published:February 25, 2019
Post category:Duchenne Muscular Dystrophy Muscular Dystrophy

The Star Gazette recently reported that Alan Ramsay Evans, a 34-year-old man from Horseheads, New York, remains in the hospital after developing respiratory failure. Three decades ago, when he was…

Curing DMD: Collaborating All Aspects of Care

Post author:Ellen Johnson
Post published:February 5, 2019
Post category:Duchenne Muscular Dystrophy Muscular Dystrophy

Duchenne muscular dystrophy (DMD) has gained recent attention. New symptom management and gene therapy treatments are at the forefront of DMD research. DMD is a genetic disease characterized by progressive…

The FDA and DMD Drug Approval – A Delicate Dance

Post author:Farrah Fontaine
Post published:October 24, 2016
Post category:Duchenne Muscular Dystrophy Rare Disease

If you, or someone you love, has Duchenne muscular dystrophy (DMD), then each new possible drug that's brought before the FDA brings with it a sense of hope. Race to…

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Do You Need Dogs for a DMD Cure?

Post author:EmpatheticBadass
Post published:August 26, 2016
Post category:Duchenne Muscular Dystrophy Rare Disease

If a treatment that would cure me or my child had to use dogs for testing, what would I do? That’s the question I had to ask when I saw…

Sarepta Advances Precision Genetic Therapies Toward Full FDA Approval: AMONDYS 45 and VYONDYS 53 Seek Traditional Approval Status

Long-Term Study Shows Givinostat’s Promise for Duchenne Muscular Dystrophy Treatment

Young Advocate: Ben’s Mission to Champion Kids’ Health in Washington, DC

A Parent’s Take on Marriage While Having a Child with Duchenne Muscular Dystrophy

First Patient Dosed in Phase 3 Study of Experimental Idiopathic Pulmonary Fibrosis Drug

Family Touched By Muscular Dystrophy Sets Up GoFundMe

Curing DMD: Collaborating All Aspects of Care

The FDA and DMD Drug Approval – A Delicate Dance

Do You Need Dogs for a DMD Cure?

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