an adeno-associated virus vector-based gene therapy indicated for the treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency. Kebilidi is the first FDA-approved gene therapy for treatment of AADC deficiency. ...
The US Food and Drug Administration approved the gene therapy valoctocogene roxaparvovec (Roctavian, BioMarin) for adults with severe hemophilia A. Valoctocogene roxaparvovec, a one-time, single-dose IV infusion, is the first gene therapy approved in the United States for severe hemophilia A...
For the first time, a gene therapy-based treatment has been given the go-ahead by regulatory authorities. China's medicines authority approved the cancer therapy after it achieved promising results in a clinical trial. The treatment, called Gendici...
WASHINGTON, Aug. 30 (Xinhua) -- The U.S. Food and Drug Administration (FDA) on Wednesday approved the country's first gene therapy that reengineers people's own immune cells to treat childhood leukemia. In a statement, the FDA called the approval "a historic action," saying it will ushe...
The U.S. Food and Drug Administration on Thursday approved the drug Elevidys (delandistrogene moxeparvovec-rokl), the first gene therapy for the treatment of children with Duchenne muscular dystrophy (DMD).
FDA approved Hemgenix (etranacogene dezaparvovec), an adeno-associated virus vector-based gene therapy for the treatment of adults with Hemophilia B
(HealthDay)—The first gene therapy has been approved to treat children younger than 2 years with spinal muscular atrophy (SMA), the U.S. Food and Drug Administration announced Friday.
The first gene therapy treatment for sickle cell disease has been approved in the U.K., a move that could offer relief to thousands of people with the disease.
The U.S. Food and Drug Administration approved Hemgenix (etranacogene dezaparvovec), an adeno-associated virus vector-based gene therapy for the treatment of adults with Hemophilia B (congenital Factor IX deficiency) who currently use Factor IX prophylaxis therapy, or have current ...
FDA: Cellular and Gene Therapy Products FDA: Oncology Center of Excellence NIH: Childhood Acute Lymphoblastic Leukemia FDA: What is Gene Therapy? FDA: Kymriah (tisagenlecleucel) product page FDA: Approved Cellular and Gene Therapy Products