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Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls.

Journal article

Darras BT. et al, (2021), N Engl J Med, 385, 427 - 435

Therapies in preclinical and clinical development for Angelman syndrome.

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Markati T. et al, (2021), Expert Opin Investig Drugs

Cholesterol metabolism is a potential therapeutic target in Duchenne muscular dystrophy.

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Amor F. et al, (2021), J Cachexia Sarcopenia Muscle

Prognostic factors and treatment-effect modifiers in spinal muscular atrophy.

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Baranello G. et al, (2021), Clin Pharmacol Ther

Risdiplam in Type 1 Spinal Muscular Atrophy.

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Baranello G. et al, (2021), N Engl J Med, 384, 915 - 923

Diagnosing X-linked Myotubular Myopathy - A German 20-year Follow Up Experience.

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Gangfuss A. et al, (2021), J Neuromuscul Dis, 8, 79 - 90

Newborn screening programs for spinal muscular atrophy worldwide: Where we stand and where to go

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Dangouloff T. et al, (2021), Neuromuscular Disorders

Natural history of Type 2 and 3 spinal muscular atrophy: 2-year NatHis-SMA study.

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Annoussamy M. et al, (2020), Ann Clin Transl Neurol

Response to letter: A decision for life - Treatment decisions in newly diagnosed families with spinal muscular atrophy.

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Kirschner J. et al, (2020), Eur J Paediatr Neurol, 30, 103 - 104

Very low residual dystrophin quantity is associated with milder dystrophinopathy.

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de Feraudy Y. et al, (2020), Ann Neurol

Novel free-circulating and extracellular vesicle-derived miRNAs dysregulated in Duchenne muscular dystrophy

Journal article

Catapano F. et al, (2020), Epigenomics, 12, 1899 - 1915

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