Galibra Neuroscience announces capsid license agreement for Gene Therapy
The SSADH Association funded the creation of a new mouse model to be created at Boston Children’s Hospital by Alexander Rotenberg, MD PhD and Henry Lee, MPhil, PhD. This mouse model allowed the researchers to turn on and off the SSADH gene mutation at different stages in the animal’s life…
Read MoreSTTR Grant to Galibra and Boston Children’s Hospital
Galibra Neuroscience has been awarded $500,000 through the U.S. Department of Health and Human Services. This grant through the Small Business Technology Transfer (STTR) program is to encourage research and development, along with the transfer of technology between small businesses and non-profit research institutions. The award has been directed to…
Read MoreDr. Rotenberg is named 2025 Harrington Scholar-Innovator Award recipient.
Congratulations to Alexander Rotenberg, MD, PhD. for being named one of ten 2025 Harrington Scholar-Innovator Award recipient by the Harrington Discovery Institute, for his project titled ‘Gene therapy for a rare inherited disease, Succinic Semialdehyde Dehydrogenase Deficiency (SSADHD)’. Dr. Rotenberg is the Director of the Neuromodulation Program at Boston Children’s…
Read MoreCelebrating Rare Disease Day
Wisconsin mother creates SSADH Association after her son is diagnosed with neuro disorder. Spectrum News Wisconsin | BY Abbey Taylor | PUBLISHED 12:59 PM ET Feb. 26, 2025 OCONOMOWOC, Wis. — A mother of three, Carolyn Hoffman said her 30-year-old middle child, Sam, was an easy baby to raise. “Sam was the…
Read MoreDr. Lee’s Podcast on Gene Therapy for SSADH Deficiency
Dr. Henry Lee, Ph.D., Boston Children’s Hospital, F.M. Kirby Neurobiology Center joins the Journal of Inherited Metabolic Disease (JIMD) podcast. Dr. Lee discusses the challenges of developing gene therapies for inherited disorders of neurotransmission. Additionally he shares the progress that his group has made with succinic semialdehyde dehydrogenase (SSADH) deficiency.…
Read MoreSSADHD Presentation at PAME
Charity Adams (Mom to Bond), Dr. Phil Pearl and Dr. Itay Tokatly Latz spoke at the 2024 PAME (Partners Against Morality in Epilepsy) event on December 5th in Los Angeles, CA. Charity spoke of the ground breaking work that Bond (pictured below) participated in just months before his passing, during…
Read More