While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...

Initiation of Phase 1b study using SGT-212 for the treatment of patients with Friedreich's Ataxia expected in 2H25. Click ...

NDAQ:SLDB) Solid Biosciences Receives FDA Fast Track Designation for SGT-212 Dual Route of Administration Gene Therapy for Friedreich's Ataxia ...

Santhera gets positive recommendation from Scottish Medicines Consortium for use of Agamree to treat Duchenne muscular dystrophy: Pratteln, Switzerland Wednesday, January 15, 2025 ...

The majority of dog owners likely view their beloved dogs as more than just loyal companions. They are practically members […] The post This New Pill Can Help Your Dog Live Longer, And Who Wouldn’t ...

The CONNECT2-EDO51 phase 2 trial looks at PGN-EDO51, an investigational therapy for Duchenne muscular dystrophy (DMD), and is still ongoing in the United Kingdom. The most-read gene therapy ...

A major supermarket has backed farmers as they protest against the Government’s planned inheritance tax raid, in a blow to Sir Keir Starmer. Sophie Throup, the head of agriculture at Morrisons ...

In Benjamin Ree's documentary, Steen's parents learn about their son's rich social life as an avid 'World of Warcraft' gamer after he dies of a degenerative neuromuscular disease.

Background: Skeletal muscle disorders associated with mutations of lamin A/C gene include autosomal Emery–Dreifuss muscular dystrophy and limb girdle muscular dystrophy 1B. The pathogenic mechanism ...

Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal dominant muscle disease with a prevalence ... Additionally, the proband was diagnosed with PPKN, an autosomal recessive disorder, due to a ...