Mar 22, 2024
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Matt Trudeau, head of ITF Therapeutics—a division of Italy's Italfarmaco—on treating Duchenne muscular dystrophy with givinostat, a histone deacetylase inhibitor.
Mar 14, 2024
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Thomas Holm Pedersen, PhD, cofounder and chief executive of NMD Pharma, a Danish company that's pursuing the use of chloride channel 1 (ClC-1) as a novel target for restoring muscle function in both myasthenia gravis and spinal muscular atrophy.
Mar 6, 2024
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Courtney Silverthorn, PhD, of the Foundation for the National Institutes of Health (FNIH) about the FNIH’s Bespoke Gene Therapy Consortium.