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What researchers published on Spinal muscular atrophy

534 papers, newest first.

Showing 20 of 534

  • Trial resultA pre-post single-arm pilot trial of a digital multicomponent intervention for self-efficacy enhancement among online support groups of primary caregivers of children with neuro-muscular disorders: SEE-NMD protocol.PloS one
  • Not labelled yetNeurofilaments as Biomarkers of Disease Activity and Treatment Response in Spinal Muscular Atrophy.European journal of neurology
  • ReviewEarly Independent Powered Mobility in Children With Severe Neuromuscular Disorders: A Scoping Review.Child: care, health and development
  • Not labelled yetDysregulation of dopamine metabolism in children with spinal muscular atrophy type 1.Brain communications
  • Not labelled yetPaediatric palliative approach in the era of disease modifying therapies in spinal muscular atrophy - a comprehensive landscape of French specialist physicians' perspectives.Orphanet journal of rare diseases
  • Not labelled yetCost-Effectiveness Analysis of Newborn Screening for Spinal Muscular Atrophy in Argentina.Value in health regional issues
  • Not labelled yetUse of European registries to characterise the changing landscape of disease progression and treatment of spinal muscular atrophy (SMA): opportunities, pitfalls and challenges.Orphanet journal of rare diseases
  • Not labelled yetPCR hyper-automation: an example of spinal muscular atrophy and severe combined immunodeficiency screening.Clinical chemistry and laboratory medicine
  • Not labelled yetBeyond motor rescue: defining adequate SMN restoration in spinal muscular atrophy.The Journal of clinical investigation
  • Not labelled yetEarly loss of axons limits the therapeutic window in a mouse model of spinal muscular atrophy.BMC neuroscience
  • Not labelled yetDefining the clinical benefits of disease-modifying therapy in people living with spinal muscular atrophy who require permanent ventilation: a UK-based modified Delphi consensus process.EClinicalMedicine
  • Not labelled yetInterval-based cycling for wheelchair users with spinal muscular atrophy: feasibility, tolerability, and exercise effects.Disability and rehabilitation. Assistive technology
  • Not labelled yetEpidural Spinal Cord Stimulation Reduces Intrinsic Gait Abnormalities in Spinal Muscular Atrophy.Muscle & nerve
  • Not labelled yetBeyond Gross Motor Function: The Association Between Fine Motor Skills and Cognitive Development in Treated SMA Type 1.Journal of clinical medicine
  • Not labelled yetCCN1-mediated stress signaling underlies intrinsic cardiomyocyte dysfunction in spinal muscular atrophy.Cell reports
  • ReviewManagement of Hip Instability in Spinal Muscular Atrophy in the Disease-Modifying Therapy Era: The "Late Autumn" or "Early Spring" of Neuro-Orthopedics?Biomedicines
  • Not labelled yetMyostatin/Smad3 in Spinal Muscular Atrophy: Biomarkers of Disease Severity and Treatment Response.Annals of clinical and translational neurology
  • ReviewCMAP as an electrophysiological biomarker in spinal muscular atrophy: A systematic review.Neurophysiologie clinique = Clinical neurophysiology
  • Randomised trialDietitian-Led Synchronous and Asynchronous Tele-Nutrition in Children With Spinal Muscular Atrophy: A Three-Arm Randomized Controlled Trial.Journal of human nutrition and dietetics : the official journal of the British Dietetic Association
  • ReviewMicrovasculopathy in spinal muscular atrophy: The endothelium as a target of SMN deficiency and an amplifier of disease.Neurobiology of disease