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Published in 2022 at "Human gene therapy"
DOI: 10.1089/hum.2022.054
Abstract: 5q-associated spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder that leads to progressive muscle atrophy and weakness. The disease is caused by a homozygous deletion or mutation in the survival of motor neuron…
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Keywords:
anti aav9;
aav9 antibodies;
adult patients;
aav9 ... See more keywords