AdvocacyLeading advocates and experts share their experiences
- 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.
- Working With Achondroplasia Advocacy Groups
Luca Santarelli, MD of Therachon discusses reaching out to achondroplasia advocacy groups. Achondroplasia is an autosomal dominant disorder characterized by dwarfism and disproportionate shortness of limbs, accompanied by life-altering complications.
- How Savara Pharmaceutical Engages Cystic Fibrosis and PAP Advocacy Groups
Rob Neville of Savara Pharmaceuticals discusses how his company engages advocacy groups in the disease they have clinical programs in, including PAP and Cystic Fibrosis.
- Jan-Anders Karlsson on Working with the Cystic Fibrosis Trust
https://www.youtube.com/watch?v=Ls0lzSOuhIM Jan-Anders Karlsson discusses Verona Pharma’s lead candidate, RPL554, as a potential therapy for cystic fibrosis (CF) patients and his company’s work with theCystic Fibrosis Trust. The Cystic Fibrosis …
- Dr. Justin Fallon of Tivorsan Pharma Discusses Biglycan and DMD Advocacy Groups
https://www.youtube.com/watch?v=9auGyomQwXU Justin Fallon, PhD is the Scientific Founder and Director of Tivorsan Pharmaceuticals, discusses his company’s plan for entering Biglycan into clinical trials and his compnay’s work with Duchenne Muscu …
- Patients at the Center of the Drug Development Process
https://www.youtube.com/watch?v=Jlxemk65rR0 Dr. John Maraganore, CEO of Alnylam Pharmaceuticals discusses how patients have become central to the drug development process.
- Jeff Marrazzo Discusses How His Company Works with Patient Groups
https://www.youtube.com/watch?v=r5iMcIjOKJ8 Jeff Marrazzo, Co-Founder and CEO of Spark Therapeutics, discusses working with advocacy groups and the importance of recognizing patients in all the work they do
- Dup15q Syndrome Overview
Vanessa Vogel-Farley, Executive Director, Dup15q Alliance discusses Dup15q syndrome, the common name for chromosome 15q11.2-q13.1 duplication syndrome. Dup15q syndrome is the common name for chromosome 15q11.2-q13.1 duplication syndrome. This is a neur …