Two Scots parents are pleading with the NHS to allow their "fighter" son access to a life changing drug before it is too late ...

Sam Millar, eight, was diagnosed with Duchenne muscular dystrophy with his parents told there was a life-changing drug that could slow the effects, but red tape means he's yet to receive it.

A multidisciplinary approach, early diagnosis, and comprehensive caregiver education are crucial in effectively managing ...

Dyne Therapeutics’ DYNE-101 shows positive results in phase 1/2 ACHIEVE study. Read why DYN stock may rise with FDA approval and a $2.78B market opportunity.

Having found that in biomedical research, the Bird Rock resident and scientist at La Jolla’s Sanford Burnham Prebys has ...

The FDA has approved an expanded indication of Soliris, an IV-administered monoclonal antibody for patients aged 6 years and ...

Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, will present data from the Phase 1/2 INSPIRE DUCHENNE ...

Jennifer Kucera has a form of muscular dystrophy that limits her ability to move. Daily caregivers help her get out of bed, ...

The Minnesota Department of Health tests for over 60 different disorders during a newborn screening, and now, two more ...

Edgewise is sponsoring an Industry Forum, “Spotlight on Becker muscular dystrophy: Understanding the lived experience of Becker and clinical advancements with a novel agent.” Only conference attendees ...

The Muscular Dystrophy Association (MDA) welcomes the U.S. Food and Drug Administration (FDA) approval of the expanded indication of Alexion/AstraZeneca’s eculizumab (Soliris) for pediatric patients ...

Edgewise Therapeutics' stock soars on positive Becker Muscular Dystrophy trial results. Learn why EWTX is a promising buy with derisked programs.