Natural History of Duchenne Muscular Dystrophy

Summary

Participation Requirements

Description

Study duration from FPFV: Q1 2019 to LPLV: Q3 2023 Primary Ojectives: To assess the natural disease course using standardized and disease appropriate evaluations over a period of 6 to 36 months in a cohort of young male subjects aged from 5 to 9 years at inclusion and diagnosed for Duchenne Muscular...

Study duration from FPFV: Q1 2019 to LPLV: Q3 2023 Primary Ojectives: To assess the natural disease course using standardized and disease appropriate evaluations over a period of 6 to 36 months in a cohort of young male subjects aged from 5 to 9 years at inclusion and diagnosed for Duchenne Muscular Dystrophy (DMD). To record a baseline period prior to the setup of an AAV gene therapy dose escalation phase I/II First in Man clinical study. Secondary Objectives: To identify clinical, imaging and/or laboratory parameters that could be predictive indicators of the disease course in DMD, within the selected range of age. To identify the best outcome measure(s) for further clinical trial assessments.

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