by Victoria Radke | Oct 29, 2021
Nolan Townsend, Chief Executive Officer of Lexeo Therapeutics, discusses the Rare Pediatric Disease designation and Orphan Drug designation granted to LX2006 for the treatment of cardiomyopathy associated with Friedreich’s ataxia. Friedreich’s ataxia is a...
by Victoria Radke | Oct 24, 2021
Joslyn Crowe, executive director of the National Niemann-Pick Disease Foundation (NNPDF), describes the Niemann-Pick community’s reaction to the FDA denying the approval of arimoclomol for the treatment of Niemann-Pick Type C (NPC). NPC is a disabling...
by Victoria Radke | Oct 20, 2021
Jörg Köhl, MD, Professor and Director at the Institute for Systemic Inflammation Research and Chairman at the Center for Infectiology and Inflammation Research Lübeck (ZIEL), discusses upcoming research on the complement system as a diagnostic tool and/or...
by Victoria Radke | Sep 7, 2021
Avanish Vellanki, Cofounder and CEO at Rain Therapeutics, gives an overview of well-differentiated and dedifferentiated liposarcoma. As Mr. Vellanki explains, liposarcoma is a rare cancer originating from fat cells located in the soft tissues of the body. It is...
by Victoria Radke | Sep 1, 2021
Karen Chen, PhD, Chief Executive Officer of the Spinal Muscular Atrophy (SMA) Foundation, discusses how the foundation is helping the SMA community expand its research. Spinal muscular atrophy (SMA) is a rare inherited neuromuscular disorder caused by an...
