FDA approves 1st gene therapy for Wiskott-Aldrich syndrome, offering a new treatment for patients lacking matched stem cell donors.
Researchers hoped they had finally found a way to get gene therapies past the blood-brain barrier. Then the first patient ...
Major is one of the first pediatric patients in Florida to receive gene therapy for sickle cell disease since the FDA ...
Using X-ray diffraction data, James Watson (1928-2025) made the seminal discovery of the double-helical structure of DNA ...
There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University's College ...
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Gene therapy wins FDA approval for life-threatening immunodeficiency
The FDA on Tuesday approved etuvetidigene autotemcel (Waskyra) as the first cell-based gene therapy for patients 6 months and ...
Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a ...
Kids and teenagers with spinal muscular atrophy had more mobility after receiving a single-dose gene replacement therapy, ...
Novartis’ Itvisma wins FDA approval as the first gene replacement therapy for spinal muscular atrophy (SMA) patients age 2 ...
The chief medical officer of Genetix Biotherapeutics, formerly known as Bluebird bio, discusses timelines and operational ...
The Greater Philadelphia Business Coalition on Health webinar series provided an in-depth framework for the advantages of including cell and gene therapy coverage in employer health plans, as well as ...
Three years after introducing Hemgenix, the first gene therapy for hemophilia B, CSL Behring has released long-term data ...
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