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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 ...
Some genetic sequences don't stay in the same place in the genome. Sometimes called jumping genes or transposons, this genetic material can hop around and rearrange itself to create new sequences.
The FDA approved an intrathecal form of Novartis’ spinal muscular atrophy gene therapy Zolgensma on Monday, broadening access ...
An international research collaboration led by Massachusetts General Hospital (MGH) investigators has identified the first gene in which mutations cause the common form of mitral valve prolapse (MVP), ...
For the first time, gene therapy is showing promise for treating inherited deafness, researchers reported Wednesday. A study involving six children born with a genetic defect that left them profoundly ...
CHICAGO (Reuters) -People who carry two copies of the APOE4 gene are virtually guaranteed to develop Alzheimer's and face symptoms at an earlier age, researchers reported on Monday in a study that ...
WASHINGTON (AP) — The first gene therapy for a deadly form of muscular dystrophy received preliminary U.S. approval on Thursday despite concerns from some government scientists about the treatment’s ...
The immune system plays a crucial role in fighting tumors and metastases. Consequently, it is decisive to conduct cancer ...
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