Rare diseases

News

After a long haul, Stealth BioTherapeutics has finally claimed FDA approval for Forzinity, the first treatment for ultra-rare disease Barth syndrome.

News

There is only one approved drug for ultra-rare disease FOP on the market, but Regeneron is hoping to add a second.

News

Servier has pushed its emerging neurology pipeline into the clinical trials stage by licensing a drug for Fragile X syndrome from the UK's Kaerus.

News

Sanofi's BTK inhibitor Wayrilz has become the first drug in the class to be approved for rare platelet-busting disease immune thrombocytopenia.

News

Ionis gets FDA nod for its HAE attack prophylactic treatment Dawnzera, offering less frequent dosing than its rivals.

News

It looks like PTC will have to carry out another trial of its drug for Friedreich ataxia before the FDA will consider approval.