Results 81 to 90 of about 4,206 (188)
Orthopaedic Surgery, EarlyView.This largest Asian case series confirms that posterior spinal fusion for SMA scoliosis is safe and effective despite severe deformity and markedly impaired pulmonary function. Meticulous multidisciplinary care, including advanced airway planning, hemodynamic support, blood management, and proactive electrolyte correction, is critical to optimizing ...
Ai Hu +7 more
wiley +1 more source
Muscle &Nerve, Volume 74, Issue 1, Page 198-206, July 2026.ABSTRACT Introduction/Aims Few studies have investigated nutrition as a primary outcome of disease modifying therapy (DMT) in spinal muscular atrophy (SMA). This study aimed to describe nutrition outcomes of DMT in children with SMA 1 and 2. Methods Children ≤ 18 years old with SMA 1 or 2 treated with DMTs, and untreated children with SMA 1 were ...
Katie O'Brien +5 more
wiley +1 more source
Nusinersen treatment of spinal muscular atrophy: current knowledge and existing gaps.
, 2018 peer reviewedSpinal muscular atrophy (SMA) is a recessive disorder caused by a mutation in the survival motor neuron 1 gene (SMN1); it affects 1 in 11 000 newborn infants.
Teresa Gidaro +3 more
core +1 more source
Nusinersen in type 0 spinal muscular atrophy: should we treat?
, 2020 A male infant affected by type 0 SMA with one copy of SMN2 received early treatment with Nusinersen at the age of 13 days. He showed mild motor improvement 2 months after treatment started but despite also showing some minimal respiratory ...
de Sanctis R. +9 more
core +1 more source
Developmental Medicine &Child Neurology, Volume 68, Issue 7, Page 909-915, July 2026.Abstract After treatment with new disease‐modifying therapies, cognitive and neurodevelopmental aspects have been observed in individuals with spinal muscular atrophy (SMA). Emerging evidence suggests that children with SMA type 1 may experience cognitive, language, and behavioural delays, with reported rates of neurodevelopmental difficulties ranging ...
Giorgia Coratti +2 more
wiley +1 more source
The impact of novel nusinersen treatment on hip stability in spinal muscular atrophy patients
, 2021 Introduction: Hip instability is common in spinal muscular atrophy (SMA) patients due to muscle imbalance. Nusinersen, a novel treatment, has documented effects on improving strength and functionality.
Wong, SHJ, Ip, HHN, Chan, HSS, To, MKT
core
Development and validation of an HILIC/MS/MS method for determination of nusinersen in rabbit plasma
HeliyonA hydrophilic interaction liquid chromatography tandem mass spectrometry (HILIC/MS/MS) method was developed and validated for the quantitative analysis of the fully phosphorothioate modified oligonucleotide nusinersen.
Xiao Zhang +6 more
doaj +1 more source
Utility of Far‐Field Potentials as a Biomarker of Neurodegeneration in Spinal Muscular Atrophy
Muscle &Nerve, Volume 73, Issue 6, Page 1089-1095, June 2026.ABSTRACT Introduction/Aims Far field potentials (FFP) have been proposed as a reliable neurophysiological prognostic biomarker in amyotrophic lateral sclerosis (ALS). This study evaluated the utility of ulnar nerve FFP as a robust research biomarker of lower motor neuron degeneration in spinal muscular atrophy (SMA).
Aicee Dawn Calma +9 more
wiley +1 more source
Muscle &Nerve, Volume 73, Issue 6, Page 1164-1171, June 2026.ABSTRACT Introduction/Aims Three disease‐modifying therapies are approved for individuals with spinal muscular atrophy (SMA); however, data concerning the combination of these therapies remain limited. This study aimed to evaluate the safety and efficacy of add‐on risdiplam in children who had experienced clinical deterioration despite gene therapy ...
Corinna Stoltenburg +4 more
wiley +1 more source
, 2020 Nusinersen is approved for the treatment of 5q spinal muscular atrophy of all types and stages in patients of all ages. Although clinical trials have shown improvements in motor function in infants and children treated with the drug, data for adults are ...
Wunderlich, Gilbert +31 more
core +1 more source