14 December 2021 - Early this month, the world's first therapy to treat spinal muscular atrophy, a rare genetic disorder ...
8 December 2021 - International report summarises drugs for rare disease best practices and recommendations. ...
7 December 2021 - Today, the FDA is issuing a draft guidance to provide recommendations for managing the administration of individualised ...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 ...
25 November 2021 - After finding out her private health insurer would no longer pay for a drug helping with ...
19 November 2021 - Today, the U.S. Food and Drug Administration approved Voxzogo (vosoritide) injection to improve growth in children five ...
12 November 2021 - NICE has today published draft guidance for public consultation which does not recommend elosulfase alfa (also called ...
4 November 2021 - The Federal Joint Committee (G-BA) today assessed the additional benefit of two active ingredients against very rare, ...
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 ...
14 September 2021 - The FDA’s approval of Aduhelm to treat Alzheimer’s disease has unleashed criticism about the decision and ...
8 September 2021 - Bylvay now approved in U.S., EU and UK as first drug treatment for patients with progressive familial intrahepatic ...
23 August 2021 - The FDA and the drug industry have jointly agreed to create several new pilot programs aimed ...
26 July 2021 - Too many Canadians are struggling to access the drugs they need, especially if they have a rare ...
21 July 2021 - Skysona is the first and only gene therapy approved in the European Union to treat early CALD. ...