ACHIEVEMENTS

• First Gene Therapy in the World for a Brain Disease (1998)
• Gene Therapy Treatment for 16 Canavan Children 
• Increased Myelin in the Brain
• Landmark Results: Atrophy in Canavan children is stopped by Gene Therapy
• Features in the Natural History Museum Exhibit: The Genome, as one of the key progress markers of the last century 
• NJ Innovation Hall of FAME: Dr. Paola Leone                                              inducted into the NJ Innovation Hall of Fame for her work on Canavan Disease

• Gene Therapy transferred to use in other degenerative diseases, such as Parkinson's 
• Medical cocktail for all Canavan children helping to increase awareness, decrease seizures and muscle tightness
• Featured on Sixty Minutes, 48 Hours, NYTimes Magazine, and beyond 
• Lifetime Achievement Award: Dr. Roger Karlin awarded by Mt. Sinai as the member of his medical school class to have most changed the face of medicine