Toward therapies for Friedreich ataxia

"Muscular Dystrophy Canada (MDC) and the Neuromuscular Disease Network for Canada (NMD4C) are pleased to invite you to a webinar on therapies for Friedreich ataxia.

This webinar is primarily targeted at Canadian clinicians, academics, and trainees with an interest in neuromuscular disease.

NMD4C and MDC are providing organizational and technical support. This is an independent clinical/academic webinar with no industry sponsorship or influence.

The NMD Rounds & Educational Webinars is a self-approved group learning activity (Section 1) as defined by the Maintenance of Certification Program of the Royal College of Physicians and Surgeons of Canada.

Chair and Speaker: Dr. Massimo Pandolfo

Agenda:

Dr. Massimo Pandolfo: Potential therapeutics for Friedreich ataxia: what is in the pipeline

Dr. David Lynch: Background in Friedreich ataxia: The role of genetics, clinical features and natural history in facilitating therapeutic development.

Learning Objectives:

1. To become familiar with the genetics, pathology, clinical features of FA.

2. To learn about the natural history of FA, outcome measures and biomarkers for use in clinical trials.

3. To know the therapeutic approaches under study for FA.

4. To understand opportunities and challenges in the development of gene therapies for FA. "