30 November 2018 - The FDA has failed to ensure that drugs given prized rare-disease status meet the intent of ...
30 November 2018 - Vickie Moored can walk again. Her words don’t slur, her vision isn’t blurred, and she no ...
28 November 2018 - The U.S. FDA today approved Firdapse (amifampridine) tablets for the treatment of Lambert-Eaton myasthenic syndrome in adults. ...
20 November 2018 - The U.S. FDA today approved Gamifant (emapalumab) for the treatment of paediatric (newborn and above) and ...
5 October 2018 - Leadiant Biosciences today announced that the FDA has granted approval to Revcovi (elapegademase-lvlr) injection in the ...
18 September 2018 - Patients with a rare disease are asking the FDA a question the agency rarely hears: Is ...
14 September 2018 - FDA's Center for Drug Evaluation and Research Director Janet Woodcock advised against a single FDA Centre of ...
21 August 2018 - Designation granted based on positive interim data from ASPIRO clinical trial. ...
20 August 2018 - EU filing accepted and under review. ...
15 August 2018 - Data from Phase 3 ARRIVAL study support treatment with Kalydeco in children ages 12 to <24 months. ...
13 August 2018 - Amicus Therapeutics on Monday set an average price of $315,000 per year for its newly approved Fabry ...
10 August 2018 - The U.S. FDA today approved Onpattro (patisiran) infusion for the treatment of peripheral nerve disease (polyneuropathy) caused ...
10 August 2018 - The U.S. FDA today approved Galafold (migalastat), the first oral medication for the treatment of adults with ...
30 July 2018 - Phase III clinical trial ongoing at multiple sites in the U.S. ...
25 June 2018 - Priority review requested to FDA. ...