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 ...
Serious side effects, including neurotoxicity and intestinal inflammation, that appear weeks or months after patients receive CAR T cell therapy for multiple myeloma share a common immune root cause, ...
On a video call in early September, Sarah Tabrizi first saw the data that she and other researchers studying Huntington’s ...
Treating our first patient is a proud moment for Abeona and a testament to the tireless resolve of our team,” said Vish ...
Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an ...
The FDA's approval of SMA treatment Itvisma marks an "important moment" for the SMA community, a Novartis executive says.
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