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Search results (28)

« Back to Publications

Rehabilitation research in spinal muscular atrophy: a call to action.

Lilien C. et al, (2025), J Neuromuscul Dis

A systematic review on motor outcome measures in congenital myopathy.

Woods M. et al, (2025), Neuromuscul Disord, 51

Pushing the boundaries: future directions in the management of spinal muscular atrophy.

Moultrie F. et al, (2025), Trends Mol Med, 31, 307 - 318

Acceptability, feasibility, safety and potential efficacy of an optimised rehabilitation for treated patients with SMA in UK: ACE SMA

Lilien C. et al, (2025), NEUROMUSCULAR DISORDERS, 53

A multicentre, prospective, longitudinal and observational natural history study for patients with nemaline myopathy in the UK with novel international collaboration

Fisher G. et al, (2025), NEUROMUSCULAR DISORDERS, 53

An algorithm for discontinuing mechanical ventilation in boys with x-linked myotubular myopathy after positive response to gene therapy: the ASPIRO experience.

Graham RJ. et al, (2024), Respir Res, 25

Patients' Perceptions of Nusinersen Effects According to Their Responder Status.

Lilien C. et al, (2024), J Clin Med, 13

Patients’ Perceptions of Nusinersen Effects according to Their Responder Status

Lilien C. et al, (2024)

X-linked myotubular myopathy: 3-year follow-up of a prospective international natural history

Seferian A. et al, (2024), NEUROMUSCULAR DISORDERS, 43

Digital outcome captures longitudinal degradation of upper-limb function in non-ambulant patients affected by neuromuscular disorders

Bancel L. et al, (2023), NEUROMUSCULAR DISORDERS, 33, S83 - S83

Patients' perceptions of the effects of Spinraza according to their status as a responder or non-responder

Lilien C. et al, (2023), NEUROMUSCULAR DISORDERS, 33, S89 - S90

Clinical Trial Readiness for Spinal Muscular Atrophy: Experience of an International Educational-Training Initiative.

Tizzano EF. et al, (2022), J Neuromuscul Dis, 9, 809 - 820

INCEPTUS Natural History, Run-in Study for Gene Replacement Clinical Trial in X-Linked Myotubular Myopathy.

Dowling JJ. et al, (2022), J Neuromuscul Dis, 9, 503 - 516

Magneto-inertial wearable device: Upper limb trajectory identification in non-ambulant patients with spinal muscular atrophy

Lilien C. et al, (2022), NEUROMUSCULAR DISORDERS, 32, S61 - S61

Functional follow-up of patients with spinal muscular atrophy treated post-symptomatically with spinraza: Clinical trial versus real life

Lilien C. et al, (2022), NEUROMUSCULAR DISORDERS, 32, S90 - S90

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