Please note that events are listed for Central Time Zone (Chicago). Upcoming Events Dec 8 Featured Featured 12:00 pm - 1:00 pm Caregivers Support Group View Calendar Recent Posts Beverly’s Journey of Hope & Giving Back Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy Fall 2024 Newsletter Experiences of mothers of long-term surviving patients with cerebral adrenoleukodystrophy: a qualitative study Gene therapy shows long-term benefit for patients with Cerebral adrenoleukodystrophy (CALD) Viking Therapeutics Announces Results from Phase 1b Clinical Trial of VK0214 in Patients with X-ALD BridgeBio Shares Positive Data from Canavan Disease Study In Memorial and Honor: August – September 2024 PMD Clinical Study Aspa Therapeutics: A Study of AAV9 Gene Therapy in Children With Canavan Disease Read More News Share your story Read Stories