The European Medicines Agency’s (EMA) human medicines committee has recommended Krystal Biotech’s Vyjuvek (beremagene ...

Approximately one to two people per million worldwide are diagnosed with the ultra-rare kidney disease every year ...

Real-time payments: US respondents place slightly higher importance on speed of payment to participants (87% US sites and 81% ...

Around the world, events are being held to mark Rare Disease Day (28 February 2025), with the aim of bringing attention to ...

Eli Lilly has unveiled plans to invest at least $27bn in building four new pharmaceutical manufacturing sites in the US.

To deliver treatments for rare diseases more quickly, companies share how they are adapting their launch strategies and ...

A study led by Queen Mary University of London has found 69 previously unidentified genetic determinants of rare diseases.

The US Food and Drug Administration (FDA) has approved Mirum Pharmaceuticals’ Ctexli (chenodiol) to treat adults with ...

The phase 3 SERENA-6 study randomised patients with HR-positive, HER2-negative advanced breast cancer whose tumours had an ...

Amgen’s Blincyto (blinatumomab) has been recommended by the National Institute for Health and Care Excellence (NICE) to treat ...

Recce Pharmaceuticals has shared positive data from a mid-stage study of its RECCE 327 topical gel (R327G) in acute bacterial ...

Johnson & Johnson (J&J) has shared positive results from a late-stage study evaluating a subcutaneous (SC) version of Tremfya ...