Risdiplam in Type 1 Spinal Muscular Atrophy
نویسندگان
چکیده
Abstract Background Type 1 spinal muscular atrophy is a rare, progressive neuromuscular disease that caused by low levels of functional survival motor neuron (SMN) protein. Risdiplam an or...
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ژورنال
عنوان ژورنال: The New England Journal of Medicine
سال: 2021
ISSN: ['0028-4793', '1533-4406']
DOI: https://doi.org/10.1056/nejmoa2009965