Rare diseases

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.

News

NICE backs NHS use of an AstraZeneca drug for one rare disease, but says it can't recommend a BioMarin therapy for another.

News

The FDA has added a warning label to bluebird bio's Skysona, after launching a probe into cases of blood cancer linked to the gene therapy.

News

Jazz Pharmaceuticals has become the first drugmaker to get FDA approval for a treatment for diffuse midline glioma with H3 K27M mutations.