Showing results (61-70 of 74) with videos related to
Sort By:
Pageof 8
Annals of Clinical and Translational Neurology|December 28, 2020
Natural history of Type 2 and 3 spinal muscular atrophy: 2-year NatHis-SMA studyMélanie Annoussamy, Andreea M Seferian, Aurore Daron, et al.Plos One|July 27, 2018
Prospective and longitudinal natural history study of patients with Type 2 and 3 spinal muscular atrophy: Baseline data NatHis-SMA studyAurélie Chabanon, Andreea Mihaela Seferian, Aurore Daron, et al.Journal of Neuromuscular Diseases|April 12, 2021
E-Health & Innovation to Overcome Barriers in Neuromuscular Diseases. Report from the 1st eNMD Congress: Nice, France, March 22-23, 2019Jonathan Pini, Gabriele Siciliano, Pauline Lahaut, et al.Neurology|March 24, 2019
X-linked myotubular myopathy: A prospective international natural history studyMélanie Annoussamy, Charlotte Lilien, Teresa Gidaro, et al.The Lancet. Neurology|December 23, 2021
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 trialEugenio Mercuri, Nicolas Deconinck, Elena S Mazzone, et al.Frontiers in Pediatrics|March 6, 2020
Palliative Care in SMA Type 1: A Prospective Multicenter French Study Based on Parents' ReportsMarie Hully, Christine Barnerias, Delphine Chabalier, et al.Orphanet Journal of Rare Diseases|December 20, 2024
Spinal muscular atrophy is also a disorder of spermatogenesisArmelle Magot, Arnaud Reignier, Olivier Binois, et al.Scientific Reports|November 29, 2024
Evidentiary basis of the first regulatory qualification of a digital primary efficacy endpointLaurent Servais, Paul Strijbos, Margaux Poleur, et al.Orphanet Journal of Rare Diseases|January 24, 2024
Multidisciplinary team meetings in treatment of spinal muscular atrophy adult patients: a real-life observatory for innovative treatmentsEmmanuelle Salort-Campana, Guilhem Solé, Armelle Magot, et al.Archives De Pediatrie : Organe Officiel De La Societe Francaise De Pediatrie|December 22, 2023
Effect of nusinersen after 3 years of treatment in 57 young children with SMA in terms of SMN2 copy number or typeFrédérique Audic, Sonia M Dubois, Julien Durigneux, et al.Pageof 8