Born in Morristown, N.J., with virtually no immune system, Cora was diagnosed with severe combined immunodeficiency, a rare ...
A cutting-edge base-edited CAR T-cell therapy is opening a revolutionary new frontier for patients battling aggressive T-cell ...
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 gene therapy for Duchenne muscular dystrophy caused complications to the liver, prompting a review of its use for younger patients. By Christina Jewett The Food and Drug Administration said on ...
A study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia offers ...
News-Medical.Net on MSN
Challenges and opportunities in delivering gene therapies for sickle cell disease and beta thalassemia
The first study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia ...
An experimental MeiraGTx gene therapy that has clinical data showing reversal of blindness in children born with a rare, inherited eye disorder is heading to Eli Lilly. According to the terms of the ...
Breakthroughs, discoveries, and DIY tips sent every weekday. Terms of Service and Privacy Policy. It might not seem so obvious when walking past rows of vacuum-sealed ...
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