The summer has been filled with debates about the safety of Elevidys, Sarepta’s gene therapy for Duchenne muscular dystrophy. Four prominent researchers are now raising a fundamental question about its efficacy: How much gene does the gene therapy actually deliver? In principle, the question should be settled. Since its first trial in 2017, Sarepta has…
USFDA Reappoints Vinay Prasad to Top Medical and Scientific Role We use cookies for analytics, advertising and to improve our site. You agree to our use of cookies by continuing to use our site. To know more, see our Cookie Policy and Cookie Settings.Ok Source link
The first child to receive a new experimental gene therapy for a rare neurological disorder died a few days after dosing, the company sponsoring that trial said Wednesday. The company, Capsida Biotherapeutics, is one of a group of companies and labs engineering new viruses potentially capable of safely ferrying genes deep into the brain. Its…
New Gene therapy trial shows restored hearing and speech in children born deaf, treated in both ears We use cookies for analytics, advertising and to improve our site. You agree to our use of cookies by continuing to use our site. To know more, see our Cookie Policy and Cookie Settings.Ok Source link
Texas Children’s is pleased to announce that a three-year-old girl has been successfully treated with the first-ever FDA-approved gene therapy treatment for AADC deficiency. Aromatic l-amino acid decarboxylase (AADC) deficiency is an extremely rare, inherited neurological disorder that prevents the brain from producing dopamine and serotonin – essential chemicals for controlling movement, mood and basic…
Many diseases are caused by a missing or defective copy of a single gene. For decades, scientists have been working on gene therapy treatments that could cure such diseases by delivering a new copy of the missing genes to the affected cells. Despite those efforts, very few gene therapy treatments have been approved by the…