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.
Barry Greene, President of Alnylam, discusses how his company works with advocacy groups in the diseases they are committed to.
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 researchers, newsmakers, patient advocates, business leaders and rare disease experts. “The discussion …
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.
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.
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).