Therapeutic approaches on CMT neuropathies
Chair: Angelo Schenone with Helena Pernice
Speaker: Kleopas Kleopa
Intro talk with discussion 30 min, followed by 5x10 min oral presentations with discussion
Objectives:
• New therapeutic approaches and drug development for inherited neuromuscular diseases, and the specific roles of the patients (as partners) and of industrial partners in the R&D process
• Value patients’ and physiotherapists’ possible contribution to the R&D processes in iNMDs.
Tasks and deliverables:
• Review and evaluate current preclinical tools and strategies for the development of new therapeutic approaches – assess the possible value of a partnership with the pharmaceutical industry
• Analyse physiotherapy and the role of patients in terms of active participation and outcome assessment, including avenues towards streamlined collection of data, anonymized in conformity with GDPR
• Assess perspectives and risks of genetic engineering methods – possible combination with current and new alternative therapeutic approaches – assess potentials raised by inclusion of patients as partners