by Peter Ciszewski | Oct 15, 2021
Matthew Klein, MD, MS, FACS, Chief Development Officer at PTC Therapeutics, discusses the results of a study evaluating intraputaminal gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency. These results were published in a recent issue of EMBO...
by Peter Ciszewski | Oct 14, 2021
Scott Fine, CEO of Cyclo Therapeutics, discusses the phase 3 trial evaluating hydroxypropyl betacyclodextrin (Trappsol Cyclo) for the treatment of Niemann-Pick disease type C1 (NPC1). NPC is a disabling neurogenetic disorder that has been diagnosed prenatally,...
by Peter Ciszewski | Oct 13, 2021
The U.S. Food and Drug Administration (FDA) has approved Rethymic (allogeneic processed thymus tissue-agdc) for the treatment of pediatric patients with congenital athymia. This is the first targeted therapy approved for this condition. Congenital athymia is a rare...
by Peter Ciszewski | Oct 13, 2021
Avanish Vellanki, Cofounder and CEO at Rain Therapeutics, discusses the role of p53 and MDM2 in cancers like liposarcoma, the mechanism of action of milademetan, and the positive pre-clinical data presented at the 2021 World Conference of Lung Cancer. As Mr. Vellanki...
by Peter Ciszewski | Oct 12, 2021
Dawn Laney, MS, CGC, CCRC, Assistant Professor, and Director of Emory Genetic Clinical Trials Center, talks about what physicians should know about Fabry disease. Fabry disease is a rare X-linked lysosomal storage disorder that results in the cellular buildup...
