In Depth 10 Jul 2024 Biotech’s battle against skeletal dysplasia: Ray of hope for children with rare genetic disorder As treatments for skeletal dysplasia are intended to alleviate painful symptoms, two drugs are battling out in the clinic to prove their effectiveness. July 10, 2024 - 7 minutesmins - By Roohi Mariam Peter Share WhatsApp Twitter Linkedin Email
News and Trends 30 Jun 2023 FDA approves BioMarin gene therapy for hemophilia A BioMarin Pharmaceutical Inc. has announced that the United States Food and Drug Administration (FDA) has approved ROCTAVIAN (valoctocogene roxaparvovec-rvox) gene therapy for the treatment of adults with severe hemophilia A (congenital factor VIII (FVIII) deficiency with FVIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test. The […] June 30, 2023 - 5 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 26 Aug 2022 First gene therapy for adults with severe hemophilia approved by EC The European Commission (EC) has granted conditional marketing authorization for a gene therapy to treat severe hemophilia A in adults it was announced yesterday (August 25). BioMarin Pharmaceutical Inc.’s Roctavian (valoctocogene roxaparvovec) treats the congenital factor 8 deficiency in adults patients without a history of factor VIII inhibitors without detectable antibodies to adeno-associated virus serotype […] August 26, 2022 - 3 minutesmins - By Liza Laws Share WhatsApp Twitter Linkedin Email
News and Trends 7 May 2020 BioMarin Partners Swiss Biotech to Develop Cardiac Gene Therapies US biotech BioMarin has partnered Swiss startup DiNAQOR to develop gene therapies to treat rare, inherited cardiac diseases. DiNAQOR will initially receive an upfront payment from BioMarin, followed by developmental, regulatory, and commercial milestone payments and a percentage of royalties on later sales. No financial details of the collaboration were revealed by either company. “We […] May 7, 2020 - 2 minutesmins - By Helen Albert Share WhatsApp Twitter Linkedin Email
News and Trends 28 Feb 2017 It’s Global Rare Disease Day! What Cures is Biotech Cooking Up? We’ve compiled a list of some of the latest advances of biotech in the field of rare disease to contribute to Rare Disease Day 2017. The European Organization for Rare Diseases (EURORDIS) is once again coordinating Rare Disease Day, which takes place on the last day of February every year. The aim is to raise awareness of […] February 28, 2017 - 5 minutesmins - By Clara Rodríguez Fernández Share WhatsApp Twitter Linkedin Email