29 June 2022 - Ipsen today announced that the U.S. FDA has accepted for priority review its resubmitted new drug ...
28 June 2022 - Approved for the treatment of the full spectrum of both late-onset Pompe disease and infantile-onset Pompe disease. ...
27 June 2022 - The Government has commissioned the Swedish Dental and Pharmaceutical Benefits Agency (TLV) to analyse and make ...
16 June 2022 - Approval based on Phase 3 trial results that demonstrated statistically significant reductions in weight and hunger in ...
15 June 2022 - Today marks the publication of the EFPIA-EURORDIS joint statement on patient access to medicines for rare diseases. ...
2 June 2022 - Radical action is needed urgently to tackle the delays patients face in getting the latest drugs for ...
20 May 2022 - Recommendation based on positive results from two clinical trials in which Xenpozyme provided improvement across multiple non-CNS ...
5 May 2022 - NICE has published evidence based recommendations on the use of selumetinib (Koselugo) for the treatment of children ...
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 ...
21 April 2022 - Beth Vanstone doesn’t want anyone else to have to fight for access to life saving medications. ...
7 April 2022 - The white paper presents an analysis of the potential risks and benefits of reforms seeking to ...
31 March 2022 - Rare Disorders NZ is extremely frustrated to hear the Minister of Health this week dismiss the call ...
28 February 2022 - England's Rare Disease Action plan is published today with details of how to improve diagnosis, care and ...
24 February 2022 - Protalix BioTherapeutics and Chiesi Global Rare Diseases today announced the submission of a marketing authorisation application via ...