Results 131 to 140 of about 4,580 (188)

Real-world treatment patterns and unmet needs in spinal muscular atrophy: a caregiver-centric survey study from China. [PDF]

BMC Neurol
Fan W, Zheng WE, Li J, Wang R, Ni S, Xue P, Xu C.   +6 more
europepmc   +1 more source

High-dose nusinersen for spinal muscular atrophy: a phase 3 randomized trial. [PDF]

Nat Med
Finkel RS, Crawford TO, Mercuri E, Sumner CJ, Garcia Romero MDM, Day JW, Montes J, Sun P, Tichler B, Paradis AD, Boesch E, Inra J, Littauer R, Sohn J, Monine M, Gambino G, Foster R, Farewell R, Fradette S.   +18 more
europepmc   +1 more source

Nusinersen rescues taurine deficiency in patients with type 1 Spinal Muscular Atrophy. [PDF]

Commun Med (Lond)
di Vito R, Hassan A, Nuzzo T, Caretto A, Panicucci C, Bruno C, Bertini E, D'Amico A, Vercelli A, Boido M, Errico F, Pellizzoni L, Usiello A.   +12 more
europepmc   +1 more source

Delphi consensus on gene therapy of spinal muscular atrophy with onasemnogene abeparvovec in Germany, Austria and Switzerland - part II - expert based recommendations for surveillance and management of side-effects. [PDF]

J Neuromuscul Dis
Ziegler A, Weiß C, Vill K, Baumann M, Bernert G, Blaschek A, Eisenkölbl A, Flotats-Bastardas M, Friese J, Holzwarth D, Ganter C, Goldhahn K, Hahn A, von der Hagen M, Hartmann H, Hasselmann O, Horber V, Husain RA, Illsinger S, Jacquier D, Johannsen J, Köhler C, Kölbel H, Kolodzig M, Klein A, Pechmann A, von Moers A, Müller-Felber W, Rauscher C, Schreiber G, Schwartz O, Sproß J, Stettner GM, Stoltenburg C, Stumpe E, Trollmann R, Wiegand G, Wilichowski E, Schara U, Kirschner J, collaborators and members of the INTEGRATE ATMP consortium.   +40 more
europepmc   +1 more source

Treatment evolution in spinal muscular atrophy: insights from the SMArtCARE registry. [PDF]

Brain
Voigt-Müller C, Pfaffenlehner M, Bernert G, Cetin H, Hagenacker T, Kölbel H, Lochmüller H, Pfeuffer C, Vill K, Walter MC, Kirschner J, Pechmann A, SMArtCARE study group .   +12 more
europepmc   +1 more source

Electrophysiological Changes in Pediatric Spinal Muscular Atrophy: Results From an Observational Study. [PDF]

Muscle Nerve
Sun R, Wang J, Han J, Jiang J, Wu J, Chen X, Deng X, Cai C, Sun D.   +8 more
europepmc   +1 more source

Correction: Matched-pair analysis of motor outcomes in adults with spinal muscular atrophy on nusinersen vs. risdiplam. [PDF]

J Neurol
Neuhoff S, Stolte B, Lipka J, Schlag M, Pul R, Schmitt LI, Leo M, Skuljec J, Deuschl C, Forsting M, Kleinschnitz C, Hagenacker T.   +11 more
europepmc   +1 more source

Editorial: Neuromuscular disorders: biomarkers, precision diagnosis, and targeted therapeutics. [PDF]

Front Neurosci
Zafarullah M, Banerjee R, Singh A, Ghosh A, Almeida S.   +4 more
europepmc   +1 more source

Serum creatinine to cystatin C ratio as a biomarker for monitoring motor-function in children with spinal muscular atrophy treated with nusinersen: a retrospective cohort study. [PDF]

BMC Neurol
Zhang YY, Wang J, Cui ZQ, Ma K.
europepmc   +1 more source

Onasemnogene Abeparvovec in Patients With SMA: Interim Results of the RESTORE Registry in Japan. [PDF]

Ann Clin Transl Neurol
Saito K, Benguerba K, Tsuchida K, Yazawa K, Tsumiyama I, Kayama H, Reyna SP, Khan F, Finkel RS.   +8 more
europepmc   +1 more source