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Publications

Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial.

Strauss KA. et al, (2022), Nature medicine

Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial.

Strauss KA. et al, (2022), Nature medicine

Financial cost and quality of life of patients with spinal muscular atrophy identified by symptoms or newborn screening.

Dangouloff T. et al, (2022), Dev Med Child Neurol

Onasemnogene abeparvovec for the treatment of spinal muscular atrophy.

McMillan HJ. et al, (2022), Expert Opin Biol Ther

Risdiplam: an investigational motor neuron-2 (SMN-2) splicing modifier for spinal muscular atrophy (SMA).

Markati T. et al, (2022), Expert opinion on investigational drugs

Respiratory management of spinal muscular atrophy type 1 patients treated with Nusinersen.

Menard J. et al, (2022), Pediatric pulmonology

Real-world and natural history data for drug evaluation in Duchenne muscular dystrophy: suitability of the North Star Ambulatory Assessment for comparisons with external controls.

Muntoni F. et al, (2022), Neuromuscul Disord

Response of plasma microRNAs to nusinersen treatment in patients with SMA

Zaharieva IT. et al, (2022), Annals of Clinical and Translational Neurology

Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial.

Mercuri E. et al, (2022), Lancet Neurol, 21, 42 - 52

Stride Velocity 95th Centile: Insights into Gaining Regulatory Qualification of the First Wearable-Derived Digital Endpoint for use in Duchenne Muscular Dystrophy Trials.

Servais L. et al, (2021), J Neuromuscul Dis

Hierarchical Bayesian modelling of disease progression to inform clinical trial design in centronuclear myopathy

Fouarge E. et al, (2021), Orphanet Journal of Rare Diseases, 16

The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophy

Scaglioni D. et al, (2021), Acta Neuropathologica Communications, 9

Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial.

Servais L. et al, (2021), Nucleic Acid Ther

Home-based gait analysis as an exploratory endpoint during a multicenter phase 1 trial in limb girdle muscular dystrophy type R2 and facioscapulohumeral muscular dystrophy.

Gidaro T. et al, (2021), Muscle Nerve

Three years pilot of spinal muscular atrophy newborn screening turned into official program in Southern Belgium.

Boemer F. et al, (2021), Sci Rep, 11

Genotype-related respiratory progression in Duchenne Muscular Dystrophy - multicentre international study.

Trucco F. et al, (2021), Muscle Nerve

Crowdfunding for neuromuscular disease treatment: the ethical implications.

Livingstone A. et al, (2021), Lancet Neurol, 20, 788 - 789

Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial.

Mercuri E. et al, (2021), Lancet Neurol, 20, 832 - 841

Upper limb disease evolution in exon 53 skipping eligible patients with Duchenne muscular dystrophy.

Lilien C. et al, (2021), Ann Clin Transl Neurol

First Regulatory Qualification of a Novel Digital Endpoint in Duchenne Muscular Dystrophy: A Multi-Stakeholder Perspective on the Impact for Patients and for Drug Development in Neuromuscular Diseases

Servais L. et al, (2021), Digital Biomarkers, 5, 183 - 190

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