Dr Essajee says that in Kenya, treatment is largely supportive: “Steroids like Deflazacort, combined with vitamin D and ...

DYNE-251 received FDA breakthrough therapy designation, showcasing its potential for patients with Duchenne muscular ...

Anaheim Ducks right wing Troy Terry is trading his hockey stick for a driver this September as he takes on a new kind of role ...

Jefferies analysts said these detailed safety outcomes confirm the gene therapy's positive risk/benefit profile in ambulatory ...

Pratteln, Switzerland, 13 August, 2025 – Santhera Pharmaceuticals (SIX: SANN) announces the signing of an exclusive agreement with Gen Ilaç ve Saglik Ürünleri San. ve Tic. A.S. (GEN) for the ...

Catalyst Pharmaceuticals stock shines with FIRDAPSE & AGAMREE growth. Learn why the biopharma name is cheap enough to start ...

Sarepta’s troubles had nothing to do with Arrowhead’s assets, and yet both companies have seen their stock prices decline ...

The shares of biotech companies fell after news that ousted regulator Vinay Prasad is set to go back to the US Food and Drug Administration. Prasad had previously overruled his own scientific review ...

Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary ...

The documentary directed by Benjamin Ree captures how Mats Steen, a young Norwegian man with muscular dystrophy, impacted ...

Last month Claire Ellis, aged 34, a teacher from Doncaster, set off from her home to cycle 100km in support of her son Declan ...

On Saturday 12 July, Claire Ellis, 34, a teacher from Doncaster set off from her home to cycle 100km in support of her son ...