Dravet Syndrome Clinical Trial With STK-001 Continues

  Barry S. Ticho, MD, PhD, Chief Medical Officer at Stoke Therapeutics, discusses the phase 1/2a MONARCH study, which is evaluating STK-001 in patients with Dravet syndrome. As Dr. Ticho explains, Dravet syndrome is a rare neurological condition that usually...

Treating Phelan-McDermid Syndrome (PMS) Is Complicated

  Alex Kolevzon, MD, Professor of Psychiatry and Pediatrics at the Icahn School of Medicine at Mount Sinai, explains the pathophysiology of Phelan-McDermid syndrome and how it is current treated. Phelan-McDermid syndrome is genetic disorder and one of few known...

Type 1 Galactosemia Roundtable Discussion

« Prev1 / 1Next »Galactosemia Roundtable DiscussionGalactosemia Foundation OverviewGalactosemia Expert PerspectivesPatient AdvocacyGalactosemia Family Story: Allison and BrooksGalactosemia Family Story: Megan and AvaGalactosemia Family Story: David and...