Kolexia
Servais Laurent Pédiatrie
Servais Laurent
Pédiatrie
Hôpital Armand Trousseau
Paris, France
383 Activités
0 Followers
Scientifique
Digital
Production scientifique
Activités par an
Expertise
Sujets de recherche
{{person.topmesh1.name}} Amyotrophie spinale Atrophie Amyotrophie Myopathie de Duchenne Dystrophies musculaires Maladies musculaires Amyotrophies spinales infantiles Myopathies congénitales structurales Maladies neuromusculaires

Collaborateurs

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Seferian Andreea
Seferian Andreea
Hôpital Pitie Salpetriere, Paris, France
Gidaro Teresa
Gidaro Teresa
Bioprojet Pharma, Paris, France
Voit Thomas
Voit Thomas
Hôpital Pitie Salpetriere, Paris, France
Vuillerot Carole
Vuillerot Carole
Hôpital Femme Mère Enfant, Bron, France

Industries

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Sarepta
2 collaboration(s)
Dernière en 2019
AveXis
2 collaboration(s)
Dernière en 2019
Biogen
1 collaboration(s)
Dernière en 2019
INC RESEARCH, LLC
1 collaboration(s)
Dernière en 2019

Dernières activités

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My trial and training journey in X-linked myotubular myopathy: mountains and valleys.
Neuromuscular disorders : NMD   26 janvier 2024
Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry.
Journal of neuromuscular diseases   18 janvier 2024
Outcome Measures in Duchenne Muscular Dystrophy: A Natural History Study: Developing Tools for Assessing the Natural History of Ambulant and Non-ambulant DMD Individuals to Assist in Antisense-oligomer Clinical Trials
Essai Clinique (University College, London)   03 janvier 2024
FIREFISH: A Two Part Seamless, Open-label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Infants With Type 1 Spinal Muscular Atrophy
Essai Clinique (Roche)   22 décembre 2023
Pathogenic DPAGT1 variants in limb-girdle congenital myasthenic syndrome (LG-CMS) associated with tubular aggregates and ORAI1 hypoglycosylation.
Neuropathology and applied neurobiology   20 décembre 2023
ASPIRO: A Phase 1/2/3, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate the Safety and Efficacy of AT132, an AAV8-Delivered Gene Therapy in X-Linked Myotubular Myopathy (XLMTM) Patients
Essai Clinique (Astellas)   19 décembre 2023
Efficacy and safety of onasemnogene abeparvovec in children with spinal muscular atrophy type 1: real-world evidence from 6 infusion centres in the United Kingdom.
The Lancet regional health. Europe   11 décembre 2023
Cost-effectiveness of spinal muscular atrophy newborn screening based on real-world data in Belgium.
Neuromuscular disorders : NMD   02 décembre 2023
Sunfish parts 1 and 2: 4-year efficacy and safety data of risdiplam in types 2 and 3 spinal muscular atrophy (SMA)
Abstracts from the World Congress of Neurology (WCN 2023)   01 décembre 2023
Letter to the Editor: In response to P.R. Clemens et al., Efficacy and Safety of Viltolarsen in Boys with Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study, and Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy.
Journal of neuromuscular diseases   22 novembre 2023