by CheckRare Staff | Jul 28, 2022
Jennifer McNary, Executive Director, Head of Patient Advocacy and Engagement at Fulcrum Therapeutics, and mother to two Duchenne muscular dystrophy patients, discusses the significance of using reachable workspace (RWS) as the primary outcome measure for REACH,...
by CheckRare Staff | Jul 12, 2022
Ricardo Dolmetsch, PhD, President of Research and Development at uniQure, discusses positive data from the low-dose cohort of the ongoing phase 1/2 clinical trial of AMT-130, the first-ever AAV gene therapy for Huntington’s disease. Huntington disease is an...
by CheckRare Staff | Jul 10, 2022
Matthew Ellinwood, DMV, PhD, Chief Scientific Officer at the MPS Society, provides an overview of the mucopolysaccharidoses (MPSs) and mucolipidoses (MLs). The MPSs are a group of inherited metabolic diseases caused by the absence or malfunctioning of certain...
by CheckRare Staff | Jun 21, 2022
Mark Forman, MD, PhD, Chief Medical Officer at Passage Bio, discusses long-term clinical and biomarker data from Cohort 1 in the phase 1/2 Imagine-1 study of PBGM01, a gene therapy under investigation for GM1 gangliosidosis. This data was presented at the 2022...
by CheckRare Staff | Jun 10, 2022
Ann Barbier, MD, PhD, Chief Medical Officer at CAMP4 Therapeutics, discusses CAMP4’s investigational RNA-based therapy for Dravet syndrome, CMP-SCN (CO-3527). Dravet syndrome is a rare neurological condition that usually appears during the first year of life as...
