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Open access

Research article

First published 2024-03-05

Risdiplam in Spinal Muscular Atrophy: Safety Profile and Use Through The Early Access to Medicine Scheme for the Paediatric Cohort in Great Britain

Nikki Cornell, Anne-Marie Childs, Elizabeth Wraige [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2024-01-02

Risdiplam Real World Data – Looking Beyond Motor Neurons and Motor Function Measures

Barbara Sitas, Mirea Hancevic, Katarina Bilic [...]

Journal of Neuromuscular Diseases

Retricted access

Meeting report

First published 2025-12

Risdiplam: A Small Molecule mRNA Splice Modifier Approved to Treat SMA Disease

Lutz Müller, Björn Jacobsen, Alexander Nürnberg

Toxicologic Pathology

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 2025-11

Pharmacokinetics of therapies approved for spinal muscular atrophy: A narrative review of current evidence

Eda Kübra Sel, Elvira Meni Maria Gkrinia, Rossana Roncato [...]

Journal of International Medical Research

Open access

Article commentary

First published 2024

Synergistic Effect of an Antisense Oligonucleotide and Small Molecule on Splicing Correction of the Spinal Muscular Atrophy Gene

Eric W Ottesen, Ravindra N Singh

Neuroscience Insights

Open access

Review article

First published 2020

The First Orally Deliverable Small Molecule for the Treatment of Spinal Muscular Atrophy

Ravindra N Singh, Eric W Ottesen, Natalia N Singh

Neuroscience Insights

Retricted access

Letter

First published 2025-4

Switching from Nusinersen to Risdiplam in Spinal Muscular Atrophy: A Comparative Analysis of Safety,Efficacy,and Economic Impact

Eleonora Castellana

Hospital Pharmacy

Retricted access

Review article

First published 2025-03

The Effect of Disease-Modifying Therapies on Lung Function and Respiratory Muscle Strength in Spinal Muscular Atrophy: Systematic Review and Meta-Analysis

Kim Kant-Smits, Bart Bartels, Laura van der Heiden [...]

Respiratory Care

Open access

Research article

First published 2024-11

Newborn screening programs for spinal muscular atrophy worldwide in 2023

Eva Vrščaj, Tamara Dangouloff, Damjan Osredkar [...]

Journal of Neuromuscular Diseases

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