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
Alnylam has revealed the data that forms the basis of its filings for its latest potential rare disease drug lumasiran, but shares dipped after an analyst suggested it could be outperformed
A pharma company’s core mission is to improve patient outcomes. This hinges on effectively influencing HCP clinical behaviour and driving disease education.