Gene Therapy Briefs: Activist Investor Reported to Take $1B Stake in BioMarin genengnews.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from genengnews.com Daily Mail and Mail on Sunday newspapers.
FDA Approves First Treatment for Patients with Rare Inherited Blood Clotting Disorder streetinsider.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from streetinsider.com Daily Mail and Mail on Sunday newspapers.
The FDA announced it has approved Adzynma as prophylactic or on-demand enzyme replacement therapy for the treatment of congenital thrombotic thrombocytopenic purpura.
The FDA approved Adzynma, the first genetically engineered protein product indicated for prophylactic (preventive) or on demand enzyme replacement therapy in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura, a rare and life-threatening blood clotting disorder.