Accelerating Drug Development for Rare Kleesfstra Syndrome with Geoff Rhyne iDefine TRANSCRIPT
Download PDFJun 16, 2026
Geoff Rhyne, Co-Founder and CEO of iDefine, discusses the mission to advance research into Kleesfstra syndrome, a rare genetic disorder, and to find a treatment where there is currently none. This organization of parents of children with KS is advocating for broader genetic sequencing to identify KS patients and...
Jun 16, 2026
Geoff Rhyne, Co-Founder and CEO of iDefine, discusses the mission to advance research into Kleesfstra syndrome, a rare genetic disorder, and to find a treatment where there is currently none. This organization of parents of children with KS is advocating for broader genetic sequencing to identify KS patients and...
Enzyme Replacement Therapy for Sanfilippo Syndrome Type B with Dr. Javier Szwarcberg Spruce Biosciences TRANSCRIPT
Download PDFMay 11, 2026
Dr. Javier Szwarcberg, CEO of Spruce Biosciences, is developing drugs for rare diseases with a primary focus on Sanfilippo Syndrome Type B, a devastating genetic neurodegenerative disease affecting children. The source of this condition is a lack of an enzyme, which results in a buildup of a toxic substance in the...
May 11, 2026
Dr. Javier Szwarcberg, CEO of Spruce Biosciences, is developing drugs for rare diseases with a primary focus on Sanfilippo Syndrome Type B, a devastating genetic neurodegenerative disease affecting children. The source of this condition is a lack of an enzyme, which results in a buildup of a toxic substance in the...
Drug Targets Iron Dysregulation in Rare Neurodegenerative Disease Multiple System Atrophy with David Stamler Alterity Therapeutics TRANSCRIPT
Download PDFMar 25, 2026
David Stamler, CEO of Alterity Therapeutics, is developing a drug to treat multiple system atrophy (MSA), a rare and rapidly progressing neurodegenerative disease that often presents as Parkinson's disease but is distinct and more aggressive. There is no single genetic cause or specific biomarker, making...