Publicaciones

improvements in walking distance during nusinersen treatment a prospective 3 year smartcare registry study

japan registry for adult subjects of spinal muscular atrophy jreact sma protocol for a longitudinal observational study

cost effectiveness analysis of gene based therapies for patients with spinal muscular atrophy type i in australia

identifying women at high risk of 90 day death after elective open abdominal aortic aneurysm repair a multicentre case control study

improved upper limb function in non ambulant children with sma type 2 and 3 during nusinersen treatment a prospective 3 years smartcare registry study

adult spinal muscular atrophy

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

effect of nusinersen on motor respiratory and bulbar function in early onset spinal muscular atrophy

distribution of weight stature and growth status in children and adolescents with spinal muscular atrophy an observational retrospective study in the united states

evolution of bulbar function in spinal muscular atrophy type 1 treated with nusinersen

real world data on access to standards of care for people with spinal muscular atrophy in the uk

clinical characteristics and misdiagnosis of spontaneous isolated superior mesenteric artery dissection

systematic literature review of clinical and economic evidence for spinal muscular atrophy

systematic literature review to assess the cost and resource use associated with spinal muscular atrophy management

systematic literature review to assess economic evaluations in spinal muscular atrophy sma

motor responses in pediatric pompe disease in the advance participant cohort

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

20 year clinical and genetic neuromuscular cohort analysis in lebanon an international effort