'Will it really work?': Young sickle cell patient among the first to start new gene therapy
Since two groundbreaking treatments were approved in late 2023, only a handful of patients have been able to access the ...
Medscape4d
Gene Therapy in Hemophilia: High Hopes and Costs, Low Uptake
Life-changing gene therapies for hemophilia have become available, yet many patients and providers have not yet rushed to try ...
Novartis gene therapy helps children with rare muscle disorder in study
Swiss drugmaker Novartis said on Monday its gene therapy helped improve motor function in children with a rare muscle ...
BioSpace7d
5 Cardio Gene Therapies on the Near Horizon
Benefiting from technological and conceptual groundwork and positive early data, gene therapies are advancing in the clinic ...