A new respiratory scoring system for evaluation of respiratory outcomes in children with spinal muscular atrophy type1 (SMA1) on SMN enhancing drugs
Sign Up to like & getrecommendations! Published in 2021 at "Neuromuscular Disorders"
DOI: 10.1016/j.nmd.2021.01.008
Abstract: Nusinersen (NUS), the first treatment approved for Spinal Muscular Atrophy type 1 (SMA1), was made available in the UK for SMA1 through the Expanded Access Program (EAP) in 2017. The Great Ormond Street Respiratory (GSR)… read more here.
Keywords: gsr; spinal muscular; treatment; sma1 ... See more keywords
Economic burden of spinal muscular atrophy in the United States: a contemporary assessment
Sign Up to like & getrecommendations! Published in 2019 at "Journal of Medical Economics"
DOI: 10.1080/13696998.2019.1646263
Abstract: Abstract Aims: To estimate healthcare resource utilization (HRU) and costs among patients with spinal muscular atrophy (SMA) type 1 (SMA1) in real-world practice, overall and among patients treated with nusinersen. As a secondary objective, HRU… read more here.
Keywords: among patients; spinal muscular; patients sma; sma1 ... See more keywords
Real-world multidisciplinary outcomes of onasemnogene abeparvovec monotherapy in patients with spinal muscular atrophy type 1: experience of the French cohort in the first three years of treatment
Sign Up to like & getrecommendations! Published in 2024 at "Orphanet Journal of Rare Diseases"
DOI: 10.1186/s13023-024-03326-3
Abstract: Spinal muscular atrophy type 1 (SMA1) is the most severe and early form of SMA, a genetic disease with motor neuron degeneration. Onasemnogene abeparvovec gene transfer therapy (GT) has changed the natural history of SMA1,… read more here.
Keywords: spinal muscular; treatment; muscular atrophy; sma1 ... See more keywords