Enhancing respiratory function in neuromuscular disease: the role of non-invasive ventilation. A narrative review. [PDF]
Banfi P+5 more
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Spinal muscular atrophy: Should we prescribe approved drugs to cohorts of patients in which they are unproven? [PDF]
Fontanelli L, Di Rauso G, Bellini G.
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Thrombospondin-4 as potential cerebrospinal fluid biomarker for therapy response in pediatric spinal muscular atrophy. [PDF]
Dobelmann V+10 more
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The Use of Whole-Body Vibration, Electrical Stimulation, and Magnetic Stimulation in Muscle Dystrophy Patients: A Scoping Review. [PDF]
Venieri A, Sarabon N.
europepmc +1 more source
Risdiplam: therapeutic effects and tolerability in a small cohort of 6 adult type 2 and type 3 SMA patients. [PDF]
Severa G+6 more
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Spinal muscular atrophy in an upper-middle-income nation before the advent of reimbursed disease-modifying therapies. [PDF]
Sakpichaisakul K+6 more
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Long-term CSF responses in adult patients with spinal muscular atrophy type 2 or 3 on treatment with nusinersen. [PDF]
Cebulla G+9 more
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Real-World Data in Children with Spinal Muscular Atrophy Type 1 on Long-Term Ventilation Receiving Gene Therapy: A Prospective Cohort Study. [PDF]
Alajjuri MA+3 more
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Profiling neuroinflammatory markers and response to nusinersen in paediatric spinal muscular atrophy. [PDF]
Zhang Q+8 more
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Neurological hospitalisations in childhood cancer survivors treated before 2001: findings from the French Childhood Cancer Survivor Study cohort. [PDF]
Rajaonera D+18 more
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