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Paper Details

Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trial.
EBioMedicine
0
2024
Des, Des-hMTM1, MTM1, MTM1 gene, Resamirigene, X-linked myotubular myopathy, XLMTM, bilparvovec, boys, congenital muscle disease, human, infant, muscle, myofibre, participants, resamirigene, resamirigene bilparvovec, respiratory insufficiency, satellite cells, skeletal muscle cells, small myofibres, weakness
Author NameAffiliation
Nancy L KuntzAnn & Robert H Lurie Children's Hospital of Chicago
Alan H BeggsThe Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School
Bryan SepulvedaInc.)
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Datasets

Release 1.2