AdvocacyLeading advocates and experts share their experiences
- 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.
- New License Agreement for the Development of Trofinetide in Rett Syndrome
Acadia Pharmaceuticals and Neuren Pharmaceuticals announced that they have entered into an exclusive North American License Agreement for the development and commercialization of trofinetide for Rett syndrome and other indications.
- The Role of Patient Advocacy in Drug Development in Cystic Fibrosis
Mr. Robert (Bob) E. Ward, Chairman of the Board and Chief Executive Officer of Eloxx Pharmaceuticals discusses the role of advocacy groups in drug development, especially for rare diseases such as cystic fibrosis (CF).
- ProQR: The Importance of Working With Rare Disease Advocacy Groups
Daniel de Boer, Founder and CEO of ProQR, discusses his company’s working relationships with various rare disease advocacy groups.
- Caregiver Action Network
John Schall is Chief Executive Officer of Caregiver Action Network (CAN) and in this video he discusses the mission of the CAN and the many valuable resources his group offers. The mission of the Caregiver Action Network is to promote resourcefulness and respect for the more than 90 million family caregivers across the country.