Rare diseases

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.

News

Novartis teams up with real-world evidence specialist Atropos Health, hoping to speed up the diagnosis and treatment of rare disease PNH.

News

Pfizer's $5.4 billion takeover of Global Blood Therapeutics three years ago is starting to look like a poor deal.

News

Precigen has won approval in the US for a first-in-class immunotherapy for recurrent respiratory papillomatosis, Papzimeos, its first product.