Essais thérapeutiques

Projets de recherche en collaboration: 

  • GNT 14 MDYF :Natural History of Duchenne Muscular DystrophyA Prospective, Interventional, Baseline Study In Young Male Subjects Aged From 5 to 9 Years
  • Biomechanical and Morphological Changes in Dystrophic Muscle (MARCHE-DMD)
  • Metabolic Exercise Testing
  • European Registry of Patients With Infantile-onset Spinal Muscular Atrophy 
  • Assessment of the Prevalence of TTR Amyloid Neuropathy in a Population of Patients With Neuropathy of Unknown Aetiology(PRETRANS) 
  • Biomark Study: Predict Intravenous Immunoglobulin Responders in Chronic Inflammatory Demyelinating Polyradiculoneuropathy(BIOMARK) .
  • Frequency of Pompe's Disease and Neuromuscular Etiologies in Patients With Restrictive Respiratory Failure Associated With Signs of Muscle Weakness (POPS3) 
  • Inflammatory Myopathies in Primary Sjögren's Syndrome (AMISS) 
  • Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease (COMET)
  • Trial to Evaluate the Efficacy and Safety of Abatacept in Combination With Standard Therapy Compared to Standard Therapy Alone in Improving Disease Activity in Adults With Active Idiopathic Inflammatory Myopathy
  • Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy
  • PHOENIX Hizentra dans les neuropathies inflammatoires. Etude de cohorte. RIPH 3.
  • BMSIM101611 A phase 3 randomized doouble blind clinical trial to evaluate the efficacy and safety of abatacept sub cutaneous with standard treatment coompared to standart treatment alone in impriving disaese activity in adults with active itdiopathic inflammatory myopathy. RIPH 1 essai thérapeutique international.