Sleep and Breathing After Nusinersen Therapy in a Child With Spinal Muscular Atrophy.
Sign Up to like & getrecommendations! Published in 2020 at "Journal of Clinical Neuromuscular Disease"
DOI: 10.1097/cnd.0000000000000304
Abstract: BACKGROUND Spinal muscular atrophy (SMA) type 3 is an autosomal recessive neurological disorder associated with a deletion/mutation in the survival motor neuron gene, with gradually progressive degeneration of the motor neurons of the spinal cord… read more here.
Keywords: therapy; sleep; spinal muscular; muscular atrophy ... See more keywords
Feasibility and safety of intrathecal treatment with nusinersen in adult patients with spinal muscular atrophy
Sign Up to like & getrecommendations! Published in 2018 at "Therapeutic Advances in Neurological Disorders"
DOI: 10.1177/1756286418803246
Abstract: Background: Nusinersen is an intrathecally administered antisense oligonucleotide (ASO) and the first approved drug for the treatment of spinal muscular atrophy (SMA). However, progressive neuromyopathic scoliosis and the presence of spondylodesis can impede lumbar punctures… read more here.
Keywords: spinal muscular; treatment; feasibility; sma type ... See more keywords
Respiratory function in adult patients with spinal muscular atrophy treated with nusinersen – a monocenter observational study
Sign Up to like & getrecommendations! Published in 2024 at "Frontiers in Neurology"
DOI: 10.3389/fneur.2024.1372674
Abstract: Background/objective Insufficiency of respiratory muscles is the most important reason for mortality in the natural history of SMA. Thus, improvement or stabilization of respiratory function by disease-modifying therapies (DMT) is a very important issue. Methods… read more here.
Keywords: sma type; type; respiratory function;
Nusinersen treatment of Spinal Muscular Atrophy Type 1 - results of expanded access programme in Poland.
Sign Up to like & getrecommendations! Published in 2021 at "Neurologia i neurochirurgia polska"
DOI: 10.5603/pjnns.a2021.0020
Abstract: AIM OF THE STUDY This study aimed to evaluate the effects of nusinersen therapy in Polish children with SMA type 1. Clinical rationale of study. Spinal muscular atrophy (SMA) is a neuromuscular disorder that is… read more here.
Keywords: atrophy; treatment; sma type; nusinersen treatment ... See more keywords