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...
Mar 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...
Targeting the Root Cause of Cystic Fibrosis Protein Dysfunction with Dr. Charlotte McKee Sionna TRANSCRIPT
Download PDFFeb 26, 2026
Dr. Charlotte McKee, Chief Medical Officer at Sionna, describes the nature of cystic fibrosis (CF, a genetic disease caused by a mutation in the single CFTR gene. While current CFTR modulator therapies do not address the most common mutation, Sionna's novel oral medicine is designed to target the previously undruggable...