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Not available anywhere: 9
Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial
E Mercuri, F Muntoni, G Baranello, R Masson, O Boespflug-Tanguy, ...
The Lancet Neurology 20 (10), 832-841, 2021
Mandates: National Institute for Health Research, UK
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 …
E Mercuri, N Deconinck, ES Mazzone, A Nascimento, M Oskoui, K Saito, ...
The Lancet Neurology 21 (1), 42-52, 2022
Mandates: National Institute for Health Research, UK, Government of Spain
Nusinersen in patients older than 7 months with spinal muscular atrophy type 1: a cohort study
K Aragon-Gawinska, AM Seferian, A Daron, E Gargaun, C Vuillerot, ...
Neurology 91 (14), e1312-e1318, 2018
Mandates: Fondazione Telethon, Italy
Emerging therapies for Duchenne muscular dystrophy
T Markati, M Oskoui, MA Farrar, T Duong, N Goemans, L Servais
The Lancet Neurology 21 (9), 814-829, 2022
Mandates: National Health and Medical Research Council, Australia, Fonds de recherche …
Dystrophin threshold level necessary for normalization of neuronal nitric oxide synthase, inducible nitric oxide synthase, and ryanodine receptor-calcium release channel type 1 …
C Gentil, C Le Guiner, S Falcone, JY Hogrel, C Peccate, S Lorain, ...
Human Gene Therapy 27 (9), 712-726, 2016
Mandates: National Institute of Health and Medical Research, France
Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophy
Conrado
J Pharmacokinet Pharmacodyn 46 (5), 441-455, 2019
Mandates: US Department of Defense, US National Institutes of Health, US Department of …
Safety and efficacy of tamoxifen in boys with Duchenne muscular dystrophy (TAMDMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
BC Henzi, S Schmidt, S Nagy, D Rubino-Nacht, S Schaedelin, ...
The Lancet Neurology 22 (10), 890-899, 2023
Mandates: Swiss National Science Foundation
Therapeutic Role of Nusinersen on Respiratory Progression in Pediatric Patients With Spinal Muscular Atrophy Type 2 and Nonambulant Type 3
F Trucco, D Ridout, H Weststrate, M Scoto, A Rohwer, G Coratti, ML Main, ...
Neurology: Clinical Practice 14 (3), e200298, 2024
Mandates: National Institute for Health Research, UK
Pathogenic DPAGT1 variants in limb‐girdle congenital myasthenic syndrome (LG‐CMS) associated with tubular aggregates and ORAI1 hypoglycosylation
L vanden Brande, S Bauché, L Pérez‐Guàrdia, D Sternberg, AM Seferian, ...
Neuropathology and Applied Neurobiology 50 (1), e12952, 2024
Mandates: National Institute of Health and Medical Research, France, Agence Nationale …
Available somewhere: 78
Nusinersen versus sham control in infantile-onset spinal muscular atrophy
RS Finkel, E Mercuri, BT Darras, AM Connolly, NL Kuntz, J Kirschner, ...
New England Journal of Medicine 377 (18), 1723-1732, 2017
Mandates: UK Medical Research Council
Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study
T Voit, H Topaloglu, V Straub, F Muntoni, N Deconinck, G Campion, ...
The Lancet Neurology 13 (10), 987-996, 2014
Mandates: UK Medical Research Council
Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy
DE Frank, FJ Schnell, C Akana, SH El-Husayni, CA Desjardins, J Morgan, ...
Neurology 94 (21), e2270-e2282, 2020
Mandates: UK Medical Research Council
Development of the P erformance of the U pper L imb module for D uchenne muscular dystrophy
A Mayhew, ES Mazzone, M Eagle, T Duong, M Ash, V Decostre, ...
Developmental Medicine & Child Neurology 55 (11), 1038-1045, 2013
Mandates: Fondazione Telethon, Italy
Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy
C Le Guiner, L Servais, M Montus, T Larcher, B Fraysse, S Moullec, ...
Nature communications 8 (1), 16105, 2017
Mandates: National Institute of Health and Medical Research, France, Fondazione …
Downregulation of myostatin pathway in neuromuscular diseases may explain challenges of anti-myostatin therapeutic approaches
V Mariot, R Joubert, C Hourdé, L Féasson, M Hanna, F Muntoni, ...
Nature communications 8 (1), 1859, 2017
Mandates: UK Medical Research Council, National Institute for Health Research, UK
Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients
C Le Guiner, M Montus, L Servais, Y Cherel, V Francois, JL Thibaud, ...
Molecular Therapy 22 (11), 1923-1935, 2014
Mandates: Fondazione Telethon, Italy
Pharmacokinetics and safety of single doses of drisapersen in non-ambulant subjects with Duchenne muscular dystrophy: results of a double-blind randomized clinical trial
KM Flanigan, T Voit, XQ Rosales, L Servais, JE Kraus, C Wardell, ...
Neuromuscular Disorders 24 (1), 16-24, 2014
Mandates: US National Institutes of Health
Distinctive serum miRNA profile in mouse models of striated muscular pathologies
N Vignier, F Amor, P Fogel, A Duvallet, J Poupiot, S Charrier, M Arock, ...
PloS one 8 (2), e55281, 2013
Mandates: National Institute of Health and Medical Research, France
Muscle MRI in patients with dysferlinopathy: pattern recognition and implications for clinical trials
J Neurol Neurosurg Psychiatry . 89 (10), 1071-1081, 2018
Mandates: UK Medical Research Council
Serum profiling identifies novel muscle miRNA and cardiomyopathy-related miRNA biomarkers in Golden Retriever muscular dystrophy dogs and Duchenne muscular dystrophy patients
L Jeanson-Leh, J Lameth, S Krimi, J Buisset, F Amor, C Le Guiner, ...
The American journal of pathology 184 (11), 2885-2898, 2014
Mandates: National Institute of Health and Medical Research, France
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