Novel Oral Therapy Targets Inflammation and Fibrosis in Pulmonary Arterial Hypertension with Sten Sörensen Cereno Scientific TRANSCRIPT
Download PDFJun 29, 2026
Sten Sörensen, CEO of Cereno Scientific, discusses work on epigenetic modulation to treat the rare disease Pulmonary Arterial Hypertension, a progressive and fatal condition for which current treatments primarily manage symptoms. Core pathological processes of PAH include inflammation, fibrosis, and the growth of...
Jun 29, 2026
Sten Sörensen, CEO of Cereno Scientific, discusses work on epigenetic modulation to treat the rare disease Pulmonary Arterial Hypertension, a progressive and fatal condition for which current treatments primarily manage symptoms. Core pathological processes of PAH include inflammation, fibrosis, and the growth of...
Accelerating Drug Development for Rare Kleefstra 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 Kleefstra 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...
Jun 16, 2026
Geoff Rhyne, Co-Founder and CEO of IDefine, discusses the mission to advance research into Kleefstra 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...
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...