Results 171 to 180 of about 7,657 (243)

Machine learning predicts treatment response to nusinersen in non-sitter Spinal Muscular Atrophy (SMA).

Stimpson G, O'Reilly E, Coratti G, Ridout D, Chakraborti T, Mitra R, de Sanctis R, Pane M, Scoto M, Mercuri E, Muntoni F, Baranello G, International SMA Consortium.   +12 more
europepmc   +1 more source

Nusinersen for spinal muscular atrophy.

Australian prescriber
openaire   +1 more source

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

New England Journal of Medicine, 2017
Richard S Finkel, Eugenio Mercuri, Nancy L Kuntz   +2 more
exaly   +2 more sources

Long-term efficacy and safety of nusinersen in adults with 5q spinal muscular atrophy: a prospective European multinational observational study

Lancet Regional Health - Europe, The
Summary Background Evidence for the efficacy of nusinersen in adults with 5q-associated spinal muscular atrophy (SMA) has been demonstrated up to a period of 16 months in relatively large cohorts but whereas patients reach a plateau over time is still to
René Günther, Claudia Diana Wurster, Svenja Brakemeier   +2 more
exaly   +2 more sources

Some of the next articles are maybe not open access.

Related searches:

Laboratory monitoring of nusinersen safety

Muscle & Nerve, 2021
AbstractIntroductionThis retrospective study reports our tertiary care center's experience with intrathecal nusinersen administration in children and adults with spinal muscular atrophy (SMA).MethodsWe reviewed safety monitoring laboratory results and need for procedural sedation and fluoroscopy‐guidance in all SMA patients receiving nusinersen between
Natalie L, Goedeker, Jennifer L, Gibbons, Arun S, Varadhachary, Anne M, Connolly, Craig M, Zaidman   +4 more
openaire   +2 more sources

Nusinersen

Reactions Weekly
  +10 more sources

An updated systematic review on spinal muscular atrophy patients treated with nusinersen, onasemnogene abeparvovec (at least 24 months), risdiplam (at least 12 months) or combination therapies.

European journal of paediatric neurology
OBJECTIVE This systematic review provides an update on outcomes for patients with spinal muscular atrophy (SMA) type 1 to 4 treated with approved therapeutics, including the most recent, risdiplam, for an observation period of up to 48 months.
Doris Giess, J. Erdos, Claudia Wild
semanticscholar   +1 more source

Long-Term Dynamics of CSF and Serum Neurofilament Light Chain in Adult Patients With 5q Spinal Muscular Atrophy Treated With Nusinersen

Neurology
Background and Objectives The availability of disease-modifying therapies for 5q-associated spinal muscular atrophy (SMA) has heightened the need to identify suitable biomarkers.
I. Cordts, C. Fuetterer, A. Wachinger, Ricarda von Heynitz, T. Kessler, M. Freigang, A. Quinten, Bogdan Bjelica, S. Brakemeier, E. Hobbiebrunken, T. Hagenacker, Susanne Petri, J. Koch, A. Hahn, P. Lingor, M. Deschauer, René Günther, M. Weiler, B. Haller, E. Feneberg   +19 more
semanticscholar   +1 more source

Differential respiratory function response in paediatric spinal muscular atrophy types 2 and 3 treated with nusinersen over 3 years.

Sleep Medicine
AIM To establish whether the initial positive effect of nusinersen (NUS) on respiratory outcomes in the first year of treatment was maintained in children with Spinal Muscular Atrophy (SMA) type 2 and to further define the effect on children with type 3 ...
A. Chacko, Peter D. Sly, Robert S. Ware, Brett Dyer, S. Deegan, Nicole M Thomas, L. Gauld   +6 more
semanticscholar   +1 more source