Rare diseases

News

Spain has agreed to reimburse Orchard Therapeutics' ex vivo gene therapy for the rare childhood disease metachromatic leukodystrophy.

News

Mirum Pharma's Ctexli has become the first FDA-approved therapy for ultra-rare disease cerebrotendinous xanthomatosis (CTX).

News

The FDA is scheduled to deliver a verdict on Sanofi and Regeneron's Dupixent for rare autoimmune skin disorder bullous pemphigoid in June.

News

The EU has approved CSL's once-monthly Andembry for hereditary angioedema, challenging therapies from Takeda and Biocryst.

News

Merck KGaA is talking to SpringWorks about a takeover, and FDA approval for a neurofibromatosis drug could have sweetened any potential deal.

News

An e-Book aimed at children with rare kidney disease aHUS can help them understand the condition, deal with emotions, and find support, says Alexion.