AdvocacyLeading advocates and experts share their experiences
- Dup15q Syndrome Overview
Vanessa Vogel-Farley, Executive Director, Dup15q Alliance discusses 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.
- Rare in Common Podcast
The 2017 Emmy-nominated Rare in Common documentary gave people from the rare disease community an opportunity to tell their stories. Today, the storytelling has expanded to the world of audio with the Rare in Common podcast.
- Cholestatic Liver Diseases: Emerging Treatment Options
Ron Cooper, President and CEO of Albireo, provides and overview of Cholestatic liver diseases. Cholestasis is a chronic condition resulting from an impairment of the biliary system, that provokes a decrease or interruption of bile secretion from the liver to the intestine, leading to the degeneration of liver tissues, chronic inflammation and the formation of scar tissue (fibrosis).
- 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.
- Eloxx Pharmaceuticals and It’s Focus on Cystic Fibrosis
Mr. Robert (Bob) E. Ward, Chairman of the Board and Chief Executive Officer of Eloxx Pharmaceuticals provides an overview of his company and it’s focus on cystic fibrosis.
- Choroideremia Overview
Cory MacDonald, Operations Manager, Choroideremia Research Foundation, discusses choroideremia, his advocacy organization, and the various clinical trials for this rare eye disorder. Cory MacDonald, Operations Manager, Choroideremia Research Foundation …
- Corbus Pharma Discusses Award from the Cystic Fibrosis Foundation
Yuval Cohen, PhD, CEO of Corbus Pharmaceuticals discusses the Development Award from the Cystic Fibrosis Foundation.