Kolexia
Laforet Pascal
Neurologie
Hôpital Raymond-Poincaré
Garches, France
446 Activités
3 Followers

Scientifique
Digital
Production scientifique
Activités par an
Expertise
Sujets de recherche
{{person.topmesh1.name}} Maladies musculaires Glycogénose de type II Glycogénose Dystrophies musculaires Cardiomyopathies Dystrophie myotonique Faiblesse musculaire Glycogénose de type III Myopathie de Duchenne

Industries

Sanofi
39 collaboration(s)
Dernière en 2023
Amicus Therapeutics
15 collaboration(s)
Dernière en 2023
Roche
13 collaboration(s)
Dernière en 2023
Biogen
6 collaboration(s)
Dernière en 2023

Dernières activités

A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD: A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease
Essai Clinique (Amicus Therapeutics)   18 mars 2024
Natural History Study in Patients With LGMDR5/2c: A Prospective Longitudinal Study of Natural History and Functional Status of Patients With Gamma-sarcoglycanopathy (LGMDR5)
Essai Clinique (Atamyo Therapeutics)   06 mars 2024
104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07).
Journal of neurology   28 février 2024
Correction to: Analysis of muscle magnetic resonance imaging of a large cohort of patient with VCP‑mediated disease reveals characteristic features useful for diagnosis.
Journal of neurology   13 février 2024
A Caribbean case of phosphoglycerate mutase deficiency: Relevance of forearm exercise test and electroneuromyography with long exercise test in the diagnosis strategy of rare muscle glycogenosis
Medical Reports   01 février 2024
METFORMYO: Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease). A Phase III, Prospective, Multicentre, Randomized, Double-blind Controlled Study
Essai Clinique (Assistance publique – Hôpitaux de Paris)   30 janvier 2024
Multidisciplinary team meetings in treatment of spinal muscular atrophy adult patients: a real-life observatory for innovative treatments.
Orphanet journal of rare diseases   24 janvier 2024
Objective evaluation of clinical actionability for genes involved in myopathies: 63 genes with a medical value for patient care
Abstracts from the 56th European Society of Human Genetics (ESHG) Conference   01 janvier 2024
OPALE: Observatoire Des Patients Atteints de Laminopathies et Emerinopathies (Observatory for PAtients With Laminopathies and Emerinopathies)
Essai Clinique (Hôpital Pitie Salpetriere)   13 décembre 2023
Identifying digital biomarkers for the self-monitoring of patients living with generalized myasthenia gravis: A proof of concept
Abstracts from the World Congress of Neurology (WCN 2023)   01 décembre 2023