Online patient communities have become a vital resource for rare disease patients, and pharma is coming round to the idea of engaging with them for real-world data.
The FDA has approved a second rare disease drug from California-based Ultragenyx in the space of a fortnight, for treatment of long-chain fatty acid oxidation disorders.
Rare disease specialist Sarepta Therapeutics has joined with Selecta Biosciences in a research pact to develop ways to allow its gene therapies to be re-dosed.
Swedish biotech Calliditas has raised $90 million in an upsized US IPO and private placement, valuing the rare kidney and liver disease specialist at $467 million as the craze for biotech I
