Results 91 to 100 of about 3,114 (145)

Open-label phase IV trial evaluating nusinersen after onasemnogene abeparvovec in children with spinal muscular atrophy. [PDF]

J Clin Invest
Proud CM, Finkel RS, Parsons JA, Masson R, Brandsema JF, Kuntz NL, Foster R, Li W, Littauer R, Sohn J, Fradette S, Youn B, Paradis AD, RESPOND Study Group.   +13 more
europepmc   +1 more source

Moving Beyond Population-Level Analyses to Characterize Individual Heterogeneity in CMAP Responses Would Enhance the Assessment of Its Clinical Utility. [PDF]

Eur J Neurol
Zhang Y, Hu J, Pu Y, Bai X.
europepmc   +1 more source

Evaluating the Effects of Nusinersen Treatment in Adults With Spinal Muscular Atrophy Using Axonal Excitability and MscanFit MUNE. [PDF]

Muscle Nerve
Alaamel A, Erdem NŞ, Gencer GYG, Kaya HC, Uysal H.   +4 more
europepmc   +1 more source

Transforming Spinal Muscular Atrophy: From Pivotal Trials to Real-World Evidence and Future Therapeutic Frontiers in Types 1 and 2. [PDF]

Biomedicines
Belančić A, Eustaquio PC, Gkrinia EMM, Rački V, Pilipović K, Vitezić D.   +5 more
europepmc   +1 more source

Nusinersen Use in Spinal Muscular Atrophy [PDF]

Pediatrics, 2019
openaire   +2 more sources

Pioneering SMA therapies for all types: survival gains, cost dynamics, and performance-based agreements. [PDF]

Cost Eff Resour Alloc
Al-Jedai A, Al-Mudaiheem H, AlSakran A, Bashiri FA, Ghamdi F, Almuhaizea MA, AlSamman A, Awad N, Ojeil R.   +8 more
europepmc   +1 more source

Advancing personalized spinal muscular atrophy care: matching the right biomarker to the right patient at the right time. [PDF]

J Neurol
Corti S, Ottoboni L, Sansone V.
europepmc   +1 more source

Respiratory function in 192 adult patients with spinal muscular atrophy (SMA) treated with nusinersen - a multicenter observational study. [PDF]

Orphanet J Rare Dis
Wurster CD, Stolte B, Kessler T, Freigang M, Bjelica B, Ilse B, Koch JC, Cordts I, Mensch A, Zeller D, Uzelac Z, Sam G, Lapp HS, Wohnrade C, Rödiger A, Muhandes MT, Schneider I, Bellut J, Nentwich J, Dorst J, Schuster J, Schreiber-Katz O, Osmanovic A, Totzeck A, Thimm A, Steinbach R, Grosskreutz J, Kleinschnitz C, Ludolph AC, Deschauer M, Kirschner J, Dreyhaupt J, Wollinsky K, Petri S, Weiler M, Hagenacker T, Günther R.   +36 more
europepmc   +1 more source

Sustainable efficacy of nusinersen in children with later-onset spinal muscular atrophy: a 3-year observational study. [PDF]

World J Pediatr
Feng YJ, Yu YC, Zhao JL, Xu L, Zhao CY, Hua Y, Sheng GX, Chen TT, Zhang Y, Wei LL, Gao F, Mao SS.   +11 more
europepmc   +1 more source

Case Report: Spinal muscular atrophy with IgA nephropathy: a coincidence or association? [PDF]

Front Pediatr
Gu Y, Wang L, Yuan X, Han Y, Han P, Cui J, Wang X, Huang Y, Zhang L, Li C.   +9 more
europepmc   +1 more source