Condition: Merosin Deficient Congenital Muscular Dystrophy · Sponsor: Institut de Myologie, France
The goal of this natural history study is to characterize the disease course, characteristics in paediatric population of LAMA2-RD (related dystrophies) patients. The aim of the study is to establish a well-described cohort of patients in France with LAMA2-RD for prospective follow-up and recruitment for future clinical trials. Participants will be follow up during a two years period regarding exhaustive aspects of the pathology: * Muscular function * Respiratory function * Cognitive phenotyping * Quality of life * Growth parameters * Biomarkers
This description comes directly from the study's public registry record.
Andreea SEFERIAN, Dr · +33 (0)1 71 73 80 50 · a.seferian@institut-myologie.org
Erwan GASNIER, PhD
Always discuss trial participation with your own doctor first.
| Centre de Référence GNMH, Pédiatrie Hôpital Raymond-Poincaré | Garches, France | Not Yet Recruiting |
| Service de MPR pédiatrique L'Escale - HCL | Lyon, France | Not Yet Recruiting |
| Département de neuropédiatrie Pôle Femme Mère Enfant CHU de Montpellier - Hôpital Gui de Chauliac | Montpellier, France | Not Yet Recruiting |
| Plateforme d'essais cliniques pédiatriques iMotion | Paris, France | Recruiting |
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Source record: clinicaltrials.gov/study/NCT06354790