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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

Free access

Research article

First published 2020

Treatment with Nusinersen – Challenges Regarding the Indication for Children with SMA Type 1

Astrid Pechmann, Matthias Baumann, Günther Bernert [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2018

Evaluation of Children with SMA Type 1 Under Treatment with Nusinersen within the Expanded Access Program in Germany

Astrid Pechmann, Thorsten Langer, David Schorling [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2024-6

Nusinersen therapy changed the natural course of spinal muscular atrophy type 1: What about spine and hip?

Niyazi Erdem Yasar, Guzelali Ozdemir, Elif Uzun Ata [...]

Journal of Children's Orthopaedics

Open access

Research article

First published 2018

An overview of the Cure SMA membership database: Highlights of key demographic and clinical characteristics of SMA members

Lisa Belter, Suzanne F. Cook, Thomas O. Crawford [...]

Journal of Neuromuscular Diseases

Free access

Research article

First published 2023

The First Report of Iranian Registry of Patients with Spinal Muscular Atrophy

Vahid Mansouri, Morteza Heidari, Maryam Bemanalizadeh [...]

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

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

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

Open access

Research article

First published 2024-04-30

Natural History of Mandibular Function in Spinal Muscular Atrophy Types 2 and 3

H. Willemijn van Bruggen, Camiel A. Wijngaarde, Faylynn Asselman [...]

Journal of Neuromuscular Diseases

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