Nusinersen for the treatment of spinal muscular atrophy
Sign Up to like & getrecommendations! Published in 2017 at "Expert Review of Neurotherapeutics"
DOI: 10.1080/14737175.2017.1364159
Abstract: ABSTRACT Introduction: Spinal muscular atrophy (SMA) is an autosomal recessive degenerative neuromuscular disorder characterized by loss of spinal motor neurons leading to muscle weakness. This review article focuses on a novel antisense oligonucleotide treatment, first… read more here.
Keywords: spinal muscular; treatment; sma; muscular atrophy ... See more keywords
Treatment preferences in spinal muscular atrophy: A swing weighting study for caregivers of patients with SMA types 1 and 2
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DOI: 10.1371/journal.pone.0309666
Abstract: Spinal muscular atrophy (SMA) is a genetic neuromuscular disorder characterized by skeletal muscle weakness and atrophy. Patients with SMA types 1 and 2 develop severe disabilities conferring substantial patient and caregiver burden. Caregiver treatment characteristic… read more here.
Keywords: spinal muscular; treatment; patients sma; sma types ... See more keywords