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