Orphan Drugs: From Regulatory Issues to Clinical Transition

Dr. Farooq’s research focuses on the identification and understanding of the molecular pathways to develop novel therapeutic strategies for neurodegenerative and neuromuscular diseases such as Spinal Muscular Atrophy (SMA), Duchene Muscular Atrophy and Myotonic Dystrophy. He is also a member for the collaborative team of clinicians/scientists at Children’s Hospital of Eastern Ontario and other pediatric centers working on initiating a dose escalation trial utilizing FDA approved drug celecoxib for the treatment of human SMA. Dr. Farooq has worked along with Dr. MacKenzie and his team and his research activities are expanding on a wider scale to repurpose drugs for the treatment of other rare disorders.

Dr. MacKenzie and his team are developing a strategic toolbox and preclinical research pathway for inherited disorders and orphan diseases. The lab has identified gene subsets for three disorders - Duchenne Muscular Dystrophy, Myotonic Dystrophy, and Spinal Muscular Atrophy - and the goal is to broaden the research to include other genetic disorders.