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improvements in walking distance during nusinersen treatment a prospective 3 year smartcare registry study
J Neuromuscul Dis
japan registry for adult subjects of spinal muscular atrophy jreact sma protocol for a longitudinal observational study
JMIR Res Protoc
cost effectiveness analysis of gene based therapies for patients with spinal muscular atrophy type i in australia
J Neurol
identifying women at high risk of 90 day death after elective open abdominal aortic aneurysm repair a multicentre case control study
EJVES Vasc Forum
improved upper limb function in non ambulant children with sma type 2 and 3 during nusinersen treatment a prospective 3 years smartcare registry study
Orphanet J Rare Dis
adult spinal muscular atrophy
Fortschr Neurol Psychiatr
survival in patients diagnosed with sma at less than 24 months of age in a population based setting before during and after introduction of nusinersen therapy experience from sweden
Eur J Paediatr Neurol
effect of nusinersen on motor respiratory and bulbar function in early onset spinal muscular atrophy
Brain
distribution of weight stature and growth status in children and adolescents with spinal muscular atrophy an observational retrospective study in the united states
Muscle Nerve
evolution of bulbar function in spinal muscular atrophy type 1 treated with nusinersen
Dev Med Child Neurol
real world data on access to standards of care for people with spinal muscular atrophy in the uk
Front Neurol
clinical characteristics and misdiagnosis of spontaneous isolated superior mesenteric artery dissection
BMC Cardiovasc Disord
systematic literature review of clinical and economic evidence for spinal muscular atrophy
Adv Ther
systematic literature review to assess the cost and resource use associated with spinal muscular atrophy management
Pharmacoeconomics
systematic literature review to assess economic evaluations in spinal muscular atrophy sma
Pharmacoeconomics
motor responses in pediatric pompe disease in the advance participant cohort
J Neuromuscul Dis
ratio of superior mesenteric artery diameter to superior mesenteric vein diameter based on non enhanced computed tomography in the early diagnosis of spontaneous isolated superior mesenteric artery dissection
World J Emerg Med
20 year clinical and genetic neuromuscular cohort analysis in lebanon an international effort
J Neuromuscul Dis