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Search results (301)

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Corrigendum to "Learnings from a registry-based cohort study for spinal muscular atrophy disease" [Neuromuscular Disorders, Volume 59, February 2026, 106332].

Jonker CJ. et al, (2026), Neuromuscul Disord

Broad lessons from negative trials in rare diseases.

Servais L. and Oskoui M., (2026), Lancet Neurol, 25, 215 - 217

Advancing treatment of spinal muscular atrophy through inhibition of the myostatin signaling pathway.

Finkel RS. et al, (2026), Expert Rev Neurother, 26, 211 - 225

A combined genomic and functional approach to One-Carbon Metabolism disorders in a population-based newborn screening

Gernez E. et al, (2026)

Real-world walking speed as a digital biomarker and outcome measure for clinical trials-a systematic review, regulatory status and future directions.

Poleur M. et al, (2026), Front Digit Health, 8

A plain language summary of the SAPPHIRE clinical trial of apitegromab in children and young adults with spinal muscular atrophy.

Crawford TO. et al, (2026), Expert Rev Neurother, 26, 101 - 120

2025 for neuromuscular diseases: a year of multiple advances.

Servais L., (2026), Lancet Neurol, 25, 22 - 24

Digital outcome measures in Duchenne muscular dystrophy: Lessons learnt from clinical trials.

González Barral C. and Servais L., (2026), J Neuromuscul Dis, 13, 77 - 85

Re-evaluating acceptable risk of death from gene therapy: A threshold study among individuals with Duchenne muscular dystrophy and their caregivers in the US and UK.

Peay H. et al, (2025), Eur J Med Genet, 78

Genomic Newborn Screening-A Long Road From Pilot to Public Policy.

Boemer F. and Servais L., (2025), JAMA Pediatr, 179, 1255 - 1256

Wearable inertial device for monitoring Parkinson's disease symptoms: a pilot study in a controlled environment.

Poleur M. et al, (2025), Sci Rep, 15

Long-Term Evaluation of Givinostat in Duchenne Muscular Dystrophy, and Natural History Comparisons.

McDonald CM. et al, (2025), Ann Clin Transl Neurol, 12, 2335 - 2348

Joint Modelling of Growth and Motor Function Centiles in Corticosteroids Treated Boys With Duchenne Muscular Dystrophy.

Stimpson G. et al, (2025), Eur J Neurol, 32

Analytical Validation of a Genomic Newborn Screening Workflow.

Hovhannesyan K. et al, (2025), Int J Neonatal Screen, 11

Genetic Newborn Screening for Retinoblastoma: A Belgian Initiative Baby Detect.

Bartoszek P. et al, (2025), Int Ophthalmol Clin, 65, 47 - 50

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