AdvocacyLeading advocates and experts share their experiences
- Working With Dravet Syndrome Advocacy Groups
Barry Ticho, MD, Chief Medical Officer at Stoke Therapeutics, discusses how his company works with Dravet syndrome advocacy groups, including the Dravet Syndrome Foundation (DSF).
- Hereditary Sensory and Autonomic Neuropathy Type 1E (HASN1E) Advocacy Group
Rachelle Dixon, President of the HSAN1E Society discusses the advocacy group she co-founded for hereditary sensory and autonomic neuropathy type 1E (HSAN1E), a progressive disorder of the central and peripheral nervous systems.
- Jenn McNarry Discusses the Sarepta AdCom and the Role of the Patient Advocate
Jennifer McNary, rare disease advocate, looks back at the Sarepta AdComm for Exondys 51 in 2016. She also discusses the role of the patient advocate.
- Hemophagocytic Lymphohistiocytosis: Advocacy Groups and Clinical Trials
Len Walt, Vice President, Head of Medical Affairs, SOBI in North America, discusses his company’s activities in Hemophagocytic lymphohistiocytosis (HLH), a rare condition in which the body makes too many activated immune cells (macrophages and lymphocytes).
- Jennifer McNary Discusses the Duchenne Muscular Dystrophy Landscape
Jennifer McNary is a consultant, speaker, and rare disease advocate. She is also a mother of two boys with Duchenne muscular dystrophy (DMD). In this video, Jennifer discusses her two boys, Max and Austin, and their journey with DMD as well as the some of the emerging therapies that other children with DMD can look forward to in the years to come.
- The Role of Advocacy Groups in Cystic Fibrosis Drug Development
Greg Duncan, President and Chief Executive Officer of Celtaxsys, discusses working with advoacy groups such as the Cystic Fibrosis Foundation, on their clinical trials.
- The Important Role of Advocacy Groups in Rare Diseases
Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City discusses the important role of advocacy groups in rare diseases.
- Choroideremia Overview
Cory MacDonald, Operations Manager, Choroideremia Research Foundation, discusses choroideremia, his advocacy organization, and the various clinical trials for this rare eye disorder.
- The Rare Disease Film Festival
Daniel DeFabio discusses the Rare Disease Film Festival, a relatively new event showcasing films from around the world which address the challenges of life with a rare disease. The first event was held in Oct of 2017 in Boston, MA.
- How Alnylam Works with Patient Advocacy Groups
Barry Greene, President of Alnylam, discusses how his company works with advocacy groups in the diseases they are committed to.