7 September 2022 - Every time I read about clinical trials testing possible treatments for rare diseases, I think of ...
31 August 2022 - Today, the US FDA approved Xenpozyme (Olipudase alfa) for intravenous infusion in paediatric and adult patients with ...
26 July 2022 - Walker Burger is beside himself with worry. ...
6 July 2022 - Kazia Therapeutics is pleased to announce that the U.S. FDA has awarded rare paediatric disease designation ...
29 June 2022 - Ipsen today announced that the U.S. FDA has accepted for priority review its resubmitted new drug ...
16 June 2022 - Approval based on Phase 3 trial results that demonstrated statistically significant reductions in weight and hunger in ...
3 May 2022 - Pliant Therapeutics announced today that PLN-74809, its oral, dual-selective αvß6/αvß1 integrin inhibitor, has received fast track ...
2 May 2022 - Orphalan announces FDA approval of Cuvrior for the treatment of adult patients with stable Wilson’s disease ...
24 January 2022 - Codexis today announced that the U.S. FDA has granted the company orphan drug designation for CDX-6512 for ...
3 January 2021 - Immix Biopharma announced today that the U.S. FDA has granted rare paediatric disease designation for IMX-110 for ...
7 December 2021 - Today, the FDA is issuing a draft guidance to provide recommendations for managing the administration of individualised ...
7 December 2021 - Cyprium Therapeutics with support from its licensing partner Sentynl Therapeutics today announced the initiation of a ...
19 November 2021 - Today, the U.S. Food and Drug Administration approved Voxzogo (vosoritide) injection to improve growth in children five ...
8 October 2021 - Today, the U.S. FDA approved Rethymic for the treatment of paediatric patients with congenital athymia, a rare ...
29 September 2021 - Mirum Pharmaceuticals today announced that the U.S. FDA has approved Livmarli (maralixibat) oral solution for the treatment ...