Risdiplam for the treatment of adults with spinal muscular atrophy: Experience of the Northern Ireland neuromuscular service-Reference-Cited by-同舟云学术

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Risdiplam for the treatment of adults with spinal muscular atrophy: Experience of the Northern Ireland neuromuscular service

Published:2022-12-05 Issue:2 Volume:67 Page:157-161
ISSN:0148-639X
Container-title:Muscle & Nerve
language:en
Short-container-title:Muscle and Nerve

Author:

McCluskey Gavin¹²^ORCID,Lamb Siobhan¹,Mason Sarah¹,NicFhirleinn Grainne³,Douglas Isobel³,Tirupathi Sandya³,Morrison Karen E.¹⁴,McConville John¹

Affiliation:

1. Department of Neurology Belfast City Hospital Belfast UK

2. Northern Ireland Centre for Stratified Medicine Altnagelvin Hospital Campus, Ulster University Derry UK

3. Department of Paediatric Neurology Royal Belfast Hospital for Sick Children Belfast UK

4. Faculty of Medicine, Health and Life Sciences Queen's University Belfast Belfast UK

Publisher

Wiley

Subject

Physiology (medical),Cellular and Molecular Neuroscience,Neurology (clinical),Physiology

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/mus.27755

Reference18 articles.

1. Spinal Muscular Atrophy

2. Risdiplam, the First Approved Small Molecule Splicing Modifier Drug as a Blueprint for Future Transformative Medicines

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1. Impact of Disease-modifying Therapies on Respiratory Function in People with Neuromuscular Disorders;Sleep Medicine Clinics;2024-09

2. Safety of risdiplam in spinal muscular atrophy patients after short‐term treatment with nusinersen;Muscle & Nerve;2024-08-13

3. Nutrition outcomes of disease modifying therapies in spinal muscular atrophy: A systematic review;Muscle & Nerve;2024-08-11

4. Clinical perspectives: Treating spinal muscular atrophy;Molecular Therapy;2024-08

5. Risdiplam therapy in adults with 5q-SMA: observational study on motor function and treatment satisfaction;BMC Neurology;2024-02-17

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