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Universal Newborn Screening for Spinal Muscular Atrophy.

Journal article

Oskoui M. et al, (2024), JAMA Pediatr

The Importance of Early Treatment of Inherited Neuromuscular Conditions.

Journal article

Mackels L. and Servais L., (2024), J Neuromuscul Dis, 11, 253 - 274

Cost-effectiveness of spinal muscular atrophy newborn screening based on real-world data in Belgium.

Journal article

Dangouloff T. et al, (2023), Neuromuscul Disord, 34, 61 - 67

The emerging spectrum of neurodevelopmental comorbidities in early-onset Spinal Muscular Atrophy.

Journal article

Baranello G. and Neurodevelopment in SMA Working Group None., (2023), Eur J Paediatr Neurol, 48, 67 - 68

Clinical and genetic features of patients suffering from CMT4J.

Journal article

Beloribi-Djefaflia S. et al, (2023), J Neurol

Titin copy number variations associated with dominant inherited phenotypes.

Journal article

Perrin A. et al, (2023), J Med Genet

First regulatory qualification of a digital primary endpoint to measure treatment efficacy in DMD.

Journal article

Servais L. et al, (2023), Nat Med, 29, 2391 - 2392

Spinal Muscular Atrophy.

Journal article

Oskoui M. and Servais L., (2023), Continuum (Minneap Minn), 29, 1564 - 1584

Tamoxifen in children with Duchenne muscular dystrophy.

Journal article

Servais L., (2023), Lancet Neurol, 22, 872 - 873

Combination disease-modifying treatment in spinal muscular atrophy: A proposed classification.

Journal article

Proud CM. et al, (2023), Ann Clin Transl Neurol

Newborn screening for spinal muscular atrophy - what have we learned?

Journal article

De Siqueira Carvalho AA. et al, (2023), Expert Rev Neurother, 1 - 8

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