The impact of three SMN2 gene copies on clinical characteristics and effect of disease-modifying treatment in patients with spinal muscular atrophy: a systematic literature review

ObjectiveTo review the clinical characteristics and effect of treatment in patients with spinal muscular atrophy (SMA) and three copies of the SMN2 gene.MethodsWe conducted a literature search in October 2022 to identify English-language clinical research on SMA that included SMN2 copy number accord...

पूर्ण विवरण

ग्रंथसूची विवरण
मुख्य लेखकों: Claudia Dosi, Riccardo Masson
स्वरूप: लेख
भाषा:English
प्रकाशित: Frontiers Media S.A. 2024-02-01
श्रृंखला:Frontiers in Neurology
विषय:
ऑनलाइन पहुंच:https://www.frontiersin.org/articles/10.3389/fneur.2024.1308296/full