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Open access

Research article

First published 2021-07-30

Oral and Swallowing Abilities Tool (OrSAT) for Type 1 SMA Patients: Development of a New Module

Beatrice Berti, Lavinia Fanelli, Roberto de Sanctis [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2023-09-08

Sex Difference in Spinal Muscular Atrophy Patients – are Males More Vulnerable?

Jianli Sun, Melissa A. Harrington, Ben Porter [...]

Journal of Neuromuscular Diseases

Free access

Research article

First published 2020

Feeding and Swallowing Problems in Infants with Spinal Muscular Atrophy Type 1: an Observational Study

A.M.B. van der Heul, I. Cuppen, R.I. Wadman [...]

Journal of Neuromuscular Diseases

Free access

Research article

First published 2018

Motor Function Test Reliability During the NeuroNEXT Spinal Muscular Atrophy Infant Biomarker Study

Kristin J. Krosschell, Michael Bosch, Leslie Nelson [...]

Journal of Neuromuscular Diseases

Free access

Research article

First published 2022-01-04

Quantitative Motion Measurements Based on Markerless 3D Full-Body Tracking in Children with SMA Highly Correlate with Standardized Motor Assessments

Astrid Blaschek, Nikolas Hesse, Birgit Warken [...]

Journal of Neuromuscular Diseases

Retricted access

Research article

First published 2022-09

Prevalence of Anti-Adeno-Associated Virus Serotype 9 Antibodies in Adult Patients with Spinal Muscular Atrophy

Benjamin Stolte, Olivia Schreiber-Katz, René Günther [...]

Human Gene Therapy

Open access

Research article

First published 2020

Infants Diagnosed with Spinal Muscular Atrophy and 4 SMN2 Copies through Newborn Screening – Opportunity or Burden? 1

Wolfgang Müller-Felber, Katharina Vill, Oliver Schwartz [...]

Journal of Neuromuscular Diseases

Free access

Research article

First published 2020

Natural History of Type 1 Spinal Muscular Atrophy in a Series of Argentinian Children

V. Aguerre, F. De Castro, J. Mozzoni [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2023-1-3

Improvements in Walking Distance during Nusinersen Treatment – A Prospective 3-year SMArtCARE Registry Study

Astrid Pechmann, Max Behrens, Katharina Dörnbrack [...]

Journal of Neuromuscular Diseases

Retricted access

Review article

First published 2023-02

Onasemnogene Abeparvovec in Type 1 Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis

Carlos Pascual-Morena, Iván Cavero-Redondo, Maribel Lucerón-Lucas-Torres [...]

Human Gene Therapy

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