What researchers published on Spinális izomatrófia (SMA)
529 papers, newest first.
Showing 20 of 529
Not labelled yetDetection of Silent Carriers of Spinal Muscular Atrophy: A Survey of Clinical Practice and Recommendations for Screening.Twin research and human genetics : the official journal of the International Society for Twin Studies
Not labelled yetIndirect treatment comparisons of nusinersen versus onasemnogene abeparvovec-brve in treatment-naïve and -experienced spinal muscular atrophy patients.Journal of medical economics
Not labelled yetQuantitative Analysis of Branaplam (LMI070) in Human Plasma by LC-MS/MS in Support of Clinical Trial in Infant Patients With Spinal Muscular Atrophy (SMA).Biomedical chromatography : BMC
Vizsgálati eredményA 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
ÁttekintésEarly 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
ÁttekintésManagement 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