Results 141 to 150 of about 4,580 (188)

Author Correction: 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

The evolving therapeutic landscape of spinal muscular atrophy - A scoping review of investigational agents, emerging delivery technologies and strategic innovations. [PDF]

Br J Clin Pharmacol
Belančić A, Eustaquio P, Gkrinia EMM, Stević I, Javor E, Lam YW, Janković S, Vitezić D.   +7 more
europepmc   +1 more source

The Dynamics of Neurofilament Light Chain in Spinal Muscular Atrophy. [PDF]

Ann Neurol
D'Silva A, Herbert K, Balaji L, He JM, Kandula T, Sampaio HA, Teoh HL, Tantsis E, Sohn J, Briggs N, Ning N, Kiernan MC, Kariyawasam DS, Farrar MA.   +13 more
europepmc   +1 more source

Economic evaluations of disease-modifying therapies for spinal muscular atrophy: a systematic literature review. [PDF]

Orphanet J Rare Dis
Yousefi M, Mehrabian A, Brown A, Butt F, Donoghue J, Parr J, Patel M, Grove A, Parsons J, Auguste P.   +9 more
europepmc   +1 more source

Patient-reported clinical characteristics and healthcare utilization in Spinal Muscular Atrophy (SMA): a cross-sectional study from Iran. [PDF]

Orphanet J Rare Dis
Montazeri B, Daroudi R, Darvishi A, Heidari M, Anabi M, Heshmat R.   +5 more
europepmc   +1 more source

Impairment of lip and tongue strength in symptomatic SMA1 patients: Results from a 4-center prospective study using the IOPI. [PDF]

J Neuromuscul Dis
Colot C, Trucco F, Ruiz Chicaiza P, Everaert E, Muys S, Tahon V, Van Himme L, Devos E, Vanlander A, Lizio A, Salmin F, Coratti G, Albamonte E, Stanca G, Pane M, Mercuri E, Sansone V, Deconinck N.   +17 more
europepmc   +1 more source

Effectiveness of Nusinersen in Type 1, 2 and 3 Spinal Muscular Atrophy: Croatian Real-World Data

Journal of Clinical Medicine, 2023
(1) Background: To investigate the real-world effectiveness and safety profile of nusinersen in Croatian paediatric and adult spinal muscular atrophy (SMA) patients.
Andrej Belančić, Dinko Vitezic, Tea Strbad   +2 more
exaly   +2 more sources

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Nusinersen

Reactions Weekly
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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, 2021
  +6 more sources