The FDA has granted fast-track designation to an investigational antisense oligonucleotide to treat individuals with myotonic ...

Dyne Therapeutics received fast-track designation from the Food and Drug Administration for a treatment of myotonic dystrophy type 1, a neuromuscular disease.

Pratteln, Switzerland, January 16, 2025 – Santhera Pharmaceuticals (SIX: SANN) announces that the National Institute for Health and Care Excellence (NICE) has issued positive Final Guidance that ...

Altruism values for treatments of rare, severe pediatric diseases have not been estimated. This study found the altruism value for a hypothetical new Duchenne muscular dystrophy treatment to be $80 ...

Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal dominant muscle disease with a prevalence of 1/10,000–1/20,000 (Lunt and Harper, 1991; Deenen et al., 2014; Tihaya et al., 2023). It is ...

Dysferlin-deficient muscular dystrophy is a devastating and untreatable disease. Using Cas9, the authors restored dysferlin in muscle stem cells from patients ex vivo and show proof-of-concept for ...

Arsenal goalscorer Ethan Nwaneri was substituted at half-time against Brighton & Hove Albion because of a muscular injury, Mikel Arteta has said. The 17-year-old opened the scoring for Arsenal in ...

offering new pathways for understanding and treating Duchenne Muscular Dystrophy (DMD). DMD, a severe genetic disorder that causes muscle weakness and shortens lifespans, arises from mutations in ...

offering new pathways for understanding and treating Duchenne Muscular Dystrophy (DMD). A groundbreaking study has shed light on the complex interactions between dystrophin, a protein critical to ...

Nila Morton was born with a rare form of muscular dystrophy called Ullrich, which requires her to rely on a wheelchair Nila Morton Nila Morton, a graduate student at Howard University, was excited ...

AGAMREE® has been recommended by NICE for treating Duchenne muscular dystrophy (DMD) in patients 4 years of age and older in England, Wales and Northern Ireland Santhera will be working closely with ...