A major trial of a promising gene therapy for Duchenne muscular dystrophy has fallen short, but researchers say the lessons learned could pave the way for better, more effective treatments.

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A new article argues dystrophin immunogenicity is an under-examined issue in the treatment of Duchenne muscular dystrophy.

None of the eteplirsen-treated patients reached a left ventricular ejection fraction below 50% compared with 22.1% of ...

Suneel Ram's caregivers share some of the lessons they have learned while caring for the 28-year-old who was diagnosed with ...

After their son was diagnosed with a life-limiting condition, one family has set themselves a huge challenge to live life to ...

Global Duchenne Muscular Dystrophy Drugs Market value attained USD 3.9 Billion in 2025. The market will achieve USD 6.5 ...

The determination comes after recruitment and dosing in certain trials were paused after a young patient died due to acute ...

Suneel Ram is a spiritual seeker, sings in a band and was the subject of a comic novel two years ago about living with Duchenne muscular dystrophy, a rare genetic ...

Learn more about whether Alvotech or Sarepta Therapeutics, Inc. is a better investment based on AAII's A+ Investor grades, which compare both companies' key financial metrics.

ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age. For patients who are ambulatory and have a confirmed mutation in the DMD gene ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, shared the following ...