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Research article

First published 2023-05-02

Longitudinal Assessment of Timed Function Tests in Ambulatory Individuals with SMA Treated with Nusinersen

Kristin J. Krosschell, Laurey Brown, Katie Hoffman [...]

Journal of Neuromuscular Diseases

Retricted access

Review article

First published 2011-12

Validation of the Expanded Hammersmith Functional Motor Scale in Spinal Muscular Atrophy Type II and III

Allan M. Glanzman, Jessica M. O’Hagen, Michael P. McDermott [...]

Journal of Child Neurology

Open access

Research article

First published 2024-04-30

Disease Trajectories in the Revised Hammersmith Scale in a Cohort of Untreated Patients with Spinal Muscular Atrophy types 2 and 3

Amy Wolfe, Georgia Stimpson, Danielle Ramsey [...]

Journal of Neuromuscular Diseases

Free access

Research article

First published 2020

Scoliosis Surgery Significantly Impacts Motor Abilities in Higher-functioning Individuals with Spinal Muscular Atrophy 1

Sally Dunaway Young, Jacqueline Montes, Rachel Salazar [...]

Journal of Neuromuscular Diseases

Free access

Research article

First published 2020

Prospective Cohort Study of Nusinersen Treatment in Adults with Spinal Muscular Atrophy

Crystal Jing Jing Yeo, Sarah D. Simeone, Elise L. Townsend [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2024-11

Comparative efficacy of risdiplam and nusinersen in Type 2 and 3 spinal muscular atrophy patients: A cohort study using real-world data

Mahmoud Reza Ashrafi, Marzieh Babaee, Seyed Saeed Hashemi Nazari [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2018

Evaluator Training and Reliability for SMA Global Nusinersen Trials 1

Allan M. Glanzman, Elena S. Mazzone, Sally Dunaway Young [...]

Journal of Neuromuscular Diseases

Free access

Research article

First published 2021-07-30

Effect of Discontinuation of Nusinersen Treatment in Long-Standing SMA3

Miriam Hiebeler, Angela Abicht, Peter Reilich [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2023-05-02

Intrathecal Onasemnogene Abeparvovec for Sitting,Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG)

Richard S. Finkel, Basil T. Darras, Jerry R. Mendell [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2018

Feasibility and safety of intrathecal treatment with nusinersen in adult patients with spinal muscular atrophy

Benjamin Stolte, Andreas Totzeck, Kathrin Kizina [...]

Therapeutic Advances in Neurological Disorders

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