AdvocacyLeading advocates and experts share their experiences
- 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.
- Current State of Duchenne Muscular Dystrophy and Future Considerations
Jennifer McNary, Rare Disease Advocate, explains why there is so much interest in Duchenne muscular dystrophy and some of the future considerations for this rare diease.
- Rare Disease Advocacy
Jodie Gillon Global Medical Lead, Patient Engagement Rare Diseases at Pfizer discusses Pfizer’s role in patient advocacy.
- 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.
- New Podcast Launched: Openly Rare with Paul Kidwell
Openly Rare with Paul Kidwell is a monthly podcast series dedicated to the discussion around rare diseases. The podcast, created and hosted by Paul Kidwell, a 20-year PR veteran within the life science industry, will feature timely conversations with r …
- Dup15q Syndrome Research
Vanessa Vogel-Farley, Executive Director, Dup15q Alliance discusses her Alliance and some of the research for Dup15q syndrome, the common name for chromosome 15q11.2-q13.1 duplication syndrome.