Search results: Sage Journals

Open accessReview articleFirst published 2021

Emerging Oligonucleotide Therapeutics for Rare Neuromuscular Diseases

Yoshitsugu Aoki, Matthew J.A. Wood

Journal of Neuromuscular Diseases

Open accessReview articleFirst published 2026

Advancing the diagnosis of rare neuromuscular and neurological diseases through the collaborative Solve-RD research framework

Lisa-Sophie Wüstner, Kornelia Ellwanger, Nika Schuermans [...]

Journal of Neuromuscular Diseases

Free accessResearch articleFirst published 2021

The Rare Knowledge Mining Methodological Framework for the Development of Practice Guidelines and Knowledge Translation Tools for Rare Diseases

C. Gagnon, J. Fortin, M.E. Lamontagne [...]

Journal of Neuromuscular Diseases

Open accessResearch articleFirst published 2026

Strengthening clinical capacity in spinal muscular atrophy: Developing and implementing training on clinical outcome assessments

Homira Osman, Maria Masnata, Zainab Adamji [...]

Journal of Neuromuscular Diseases

Open accessResearch articleFirst published 2026

Could an outcome-based agreement be operationalized using real-world data from the Canadian Neuromuscular Disease Registry? Perspectives from an expert-led assessment in spinal muscular atrophy

Arif Mitha, Victoria Hodgkinson, Susi Vander Wyk [...]

Therapeutic Advances in Rare Disease

Open accessReview articleFirst published May, 2025

A state-of-the-art review of registries in spinal muscular atrophy: A valuable resource for clinical research

Lakshmi Balaji, Michelle A Farrar, Eppie M Yiu [...]

Journal of Neuromuscular Diseases

Available accessResearch articleFirst published October, 2019

Sugammadex is changing the paradigm in neuromuscular blockade in patients with myotonic dystrophy

Joana Teixeira, Bernardo Matias, Irene Ferreira [...]

Journal of Perioperative Practice

Open accessResearch articleFirst published 2025

Efficacy of respiratory muscle training in improving pulmonary function and survival in patients with amyotrophic lateral sclerosis: a systematic review and meta-analysis

María Jesús Benzo-Iglesias, Patricia Rocamora-Pérez, María de los Ángeles Valverde-Martínez [...]

Therapeutic Advances in Respiratory Disease

Restricted accessResearch articleFirst published September, 1985

Bronchomalacia in the Neonate

James C. Denneny

Annals of Otology,Rhinology & Laryngology

Open accessMeeting reportFirst published July 30, 2021

E-Health & Innovation to Overcome Barriers in Neuromuscular Diseases. Report from the 1st eNMD Congress: Nice,France,March 22-23,2019

Jonathan Pini, Gabriele Siciliano, Pauline Lahaut [...]

Journal of Neuromuscular Diseases

Open accessReview articleFirst published 2021

Emerging Oligonucleotide Therapeutics for Rare Neuromuscular Diseases

Yoshitsugu Aoki, Matthew J.A. Wood

Journal of Neuromuscular Diseases

Open accessReview articleFirst published 2026

Advancing the diagnosis of rare neuromuscular and neurological diseases through the collaborative Solve-RD research framework

Lisa-Sophie Wüstner, Kornelia Ellwanger, Nika Schuermans [...]

Journal of Neuromuscular Diseases

Free accessResearch articleFirst published 2021

The Rare Knowledge Mining Methodological Framework for the Development of Practice Guidelines and Knowledge Translation Tools for Rare Diseases

C. Gagnon, J. Fortin, M.E. Lamontagne [...]

Journal of Neuromuscular Diseases

Open accessResearch articleFirst published 2026

Strengthening clinical capacity in spinal muscular atrophy: Developing and implementing training on clinical outcome assessments

Homira Osman, Maria Masnata, Zainab Adamji [...]

Journal of Neuromuscular Diseases

Open accessResearch articleFirst published 2026

Could an outcome-based agreement be operationalized using real-world data from the Canadian Neuromuscular Disease Registry? Perspectives from an expert-led assessment in spinal muscular atrophy

Arif Mitha, Victoria Hodgkinson, Susi Vander Wyk [...]

Therapeutic Advances in Rare Disease

Open accessReview articleFirst published May, 2025

A state-of-the-art review of registries in spinal muscular atrophy: A valuable resource for clinical research

Lakshmi Balaji, Michelle A Farrar, Eppie M Yiu [...]

Journal of Neuromuscular Diseases

Available accessResearch articleFirst published October, 2019

Sugammadex is changing the paradigm in neuromuscular blockade in patients with myotonic dystrophy

Joana Teixeira, Bernardo Matias, Irene Ferreira [...]

Journal of Perioperative Practice

Open accessResearch articleFirst published 2025

Efficacy of respiratory muscle training in improving pulmonary function and survival in patients with amyotrophic lateral sclerosis: a systematic review and meta-analysis

María Jesús Benzo-Iglesias, Patricia Rocamora-Pérez, María de los Ángeles Valverde-Martínez [...]

Therapeutic Advances in Respiratory Disease

Restricted accessResearch articleFirst published September, 1985

Bronchomalacia in the Neonate

James C. Denneny

Annals of Otology,Rhinology & Laryngology

Open accessMeeting reportFirst published July 30, 2021

E-Health & Innovation to Overcome Barriers in Neuromuscular Diseases. Report from the 1st eNMD Congress: Nice,France,March 22-23,2019

Jonathan Pini, Gabriele Siciliano, Pauline Lahaut [...]

Journal of Neuromuscular Diseases