On a video call in early September, Sarah Tabrizi first saw the data that she and other researchers studying Huntington’s ...
Born in Morristown, N.J., with virtually no immune system, Cora was diagnosed with severe combined immunodeficiency, a rare ...
A single-dose gene replacement therapy is found to improve movement ability in children over 2 years of age and teenagers with spinal muscular atrophy, according to research published in Nature ...
Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced the first commercial patient treatment with FDA-approved ZEVASKYN ...
Almost two thirds of patients with a rare form of blood cancer who took part in a clinical trial of a revolutionary new ...
Itvisma contains the active ingredient in Zolgensma approved in 2019 for children younger than 2 years but is reformulated ...
Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an ...
Irish Independent on MSN
Innovative therapy gives hope to patients whose blood cancer ‘seemed incurable’
Almost two thirds of patients with T-cell acute lymphoblastic leukaemia involved in a clinical trial of the treatment are now ...
MedPage Today on MSN
FDA Gives Broad Approval to Gene Therapy for Rare Neurologic Disease
The FDA approved the intrathecal gene therapy onasemnogene abeparvovec (Itvisma) to treat spinal muscular atrophy (SMA) in ...
A groundbreaking new treatment using genome-edited immune cells, developed by scientists at UCL (University College London) and Great Ormond Street ...
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Patients with previously untreatable blood cancer now disease-free after world-first gene therapy
Patients with a previously untreatable form of blood cancer are now living disease-free after a world-first gene therapy. Alyssa Tapley, 16, became the first patient with an aggressive form of ...
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