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Articles (210)

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1-10 of 210 results for Spinal muscular atrophy (SMA)

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Select article: Onasemnogene Abeparvovec: Post-infusion Efficacy and Safety in Patients With Spinal Muscular Atrophy (SMA)—A Fondazione Policlinico Gemelli IRCCS Experience

Open access

Research article

First published 2024-2

Onasemnogene Abeparvovec: Post-infusion Efficacy and Safety in Patients With Spinal Muscular Atrophy (SMA)—A Fondazione Policlinico Gemelli IRCCS Experience

Michele Favia, Domenico Tarantino, Lidia Di Cerbo [...]

Hospital Pharmacy

Select article: Cost of illness of spinal muscular atrophy (SMA) in Italy

Open access

Research article

First published 2019

Cost of illness of spinal muscular atrophy (SMA) in Italy

Andrea Marcellusi, Chiara Bini, Jacopo Casiraghi [...]

Global & Regional Health Technology Assessment: Italian;Northern Europe and Spanish

Select article: Burden of Spinal Muscular Atrophy (SMA) on Patients and Caregivers in Canada

Open access

Research article

First published 2021-07-30

Burden of Spinal Muscular Atrophy (SMA) on Patients and Caregivers in Canada

H.J. McMillan, B. Gerber, T. Cowling [...]

Journal of Neuromuscular Diseases

Select article: Spinal Muscular Atrophy (SMA) Subtype Concordance in Siblings: Findings From the Cure SMA Cohort

Open access

Research article

First published 2020

Spinal Muscular Atrophy (SMA) Subtype Concordance in Siblings: Findings From the Cure SMA Cohort

Cynthia C. Jones, Suzanne F. Cook, Jill Jarecki [...]

Journal of Neuromuscular Diseases

Select article: REGISTRE SMA FRANCE: A nationwide observational registry of patients with spinal muscular atrophy in France

Open access

Research article

First published 2025-11

REGISTRE SMA FRANCE: A nationwide observational registry of patients with spinal muscular atrophy in France

Lamiae Grimaldi, Rocio Garcia-Uzquiano, Marta Gomez-Garcia de la Banda [...]

Journal of Neuromuscular Diseases

Select article: Development of the SMA EFFORT: A new approach to characterize perceived physical fatigability in spinal muscular atrophy

Open access

Research article

First published 2025-1

Development of the SMA EFFORT: A new approach to characterize perceived physical fatigability in spinal muscular atrophy

Rafael Rodriguez-Torres, Cara H Kanner, Emma L Gay [...]

Journal of Neuromuscular Diseases

Select article: The Cure SMA Membership Surveys: Highlights of Key Demographic and Clinical Characteristics of Individuals with Spinal Muscular Atrophy

Open access

Research article

First published 2021

The Cure SMA Membership Surveys: Highlights of Key Demographic and Clinical Characteristics of Individuals with Spinal Muscular Atrophy

Lisa Belter, Jill Jarecki, Sandra P. Reyna [...]

Journal of Neuromuscular Diseases

Select article: Patient-reported assessment of bulbar function in spinal muscular atrophy (SMA): Validation of a self-report scale

Open access

Research article

First published 2025-5

Patient-reported assessment of bulbar function in spinal muscular atrophy (SMA): Validation of a self-report scale

Maureen A Lefton-Greif, Lisa Belter, Jill Jarecki [...]

Journal of Neuromuscular Diseases

Select article: A Systematic Literature Review of the Natural History of Respiratory,Swallowing,Feeding,and Speech Functions in Spinal Muscular Atrophy (SMA)

Open access

Review article

First published 2024

A Systematic Literature Review of the Natural History of Respiratory,Swallowing,Feeding,and Speech Functions in Spinal Muscular Atrophy (SMA)

Yasmina Martí, Valerie Aponte Ribero, Sarah Batson [...]

Journal of Neuromuscular Diseases

Select article: ZPR1-Dependent Neurodegeneration Is Mediated by the JNK Signaling Pathway

Open access

Research article

First published 2019

ZPR1-Dependent Neurodegeneration Is Mediated by the JNK Signaling Pathway

Xiaoting Jiang, Annapoorna Kannan, Laxman Gangwani

Neuroscience Insights

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