Johnny Quintana’s life changed at the age of 19 when he was diagnosed with a rare form of muscular dystrophy called ...

Teen Mom star Leah Messer admits it’s “very challenging” helping daughter Aliannah “balance” her desire for freedom with the ...

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

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 ...

Vamorolone (Agamree, Santhera) is indicated for 'the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older'. The anticipated list price of vamorolone is £4,585.87 per 100 ...

Muscular dystrophy can appear in infancy up to middle age or later, and its form and severity are determined in part by the age at which it occurs. Some types of muscular dystrophy typically ...

Dystrophy is any condition in which a part of the body weakens or wastes away. In muscular dystrophy, the weakness is in the muscles. An inherited genetic mistake prevents the body from making a ...

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 ...

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 ...