Methodology, choice of design and writing of a procedure of First in Man - pivotal, gene-therapy study in children with Duchenne Muscular Dystrophy. Discussions with Regulatory agencies (EMA/FDA)
Participation in writing, choice of designs, within the framework of protocols for extensions of Smecta indication (new countries, new indications, new form of administration).
Biostatistics support (main biostatistician) throughout the development program (phases II - dose selection, phases III - demonstration of efficiency, participation in advices and support for submission to EMA).
Creation of a composite criterion combining the signs and symptoms of the pathology.
Obtaining the MMA from the European Commission on 03/25/2015 (first cyclosporine authorized in this context). Participation in scientific publications.
Assessing of the effectiveness of antiparasitics drugs for dog caused by an infection due to mosquitoes. Development of the methodology for statistical analysis of the experimental plan, results interpretation.
Biostatistics assistance for the analysis of the 2 French confirmatory clinical trials. Statistical methodology, in particular on missing data imputation. Participation in the development of the dossier with the ANSM (French National Agency for the safety of medicines and Health products).