AdvocacyLeading advocates and experts share their experiences
- 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 …
- Summit Therapeutics Discusses It’s Multiple DMD Partnerships
https://www.youtube.com/watch?v=WiK5qFBg6ro Glyn Edwards of Summit Therapeutics discusses his company’s partnerships with the Duchenne muscular dystrophy (DMD) community and with Sarepta Therapeutics. DMD is one of the most common, fatal genetic disord …
- Being Diagnosed with Charcot-Marie-Tooth Disease
Charcot-Marie-Tooth disease (CMT) is one of the most common inherited neurological disorders, affecting approximately 1 in 2,500 people in the United States. The disease is named for the three physicians who first identified it in 1886 – Jean-Martin Ch …
- Stefan Weber Discusses How His Company Works With Rett Syndrome Advocacy Groups
https://www.youtube.com/watch?v=TUfrCqDmt8o Stefan Weber, CEO of Newron Pharmaceuticals discusses how his company works with Rett Syndrome advocacy groups. Newron is focused on the development of novel therapies for patients with diseases of the centra …
- Chris Garabedian Discusses Interacting With Duchenne Patients and Advocates
https://www.youtube.com/watch?v=B2hlbRQqTVc Chris Garabedian Discusses Interacting With Duchenne muscular dystryphy Patients and Advocates.
- Thijs Spoor of AzurRx Provides an Overview of Cystic Fibrosis
Thijs Spoor of AzurRx Provides an Overview of Cystic Fibrosis Cystic fibrosis (CF) is a life-threatening genetic disease that primarily affects the lungs and digestive system. An estimated 30,000 children and adults in the United States (70,000 worldwi …
- The Importance of Advocacy Groups Such as the Fragile X Foundation
Dr. Daniel Alkon of Neurotrope BioSciences discusses the importance of partnering with Advocacy Groups such as the Fragile X Foundation for company. Neurotrope has reached out to advocacy groups for their clinical trial strategy for Bryostatin 1 for th …
- Rare Disease Advocacy Groups
Sam Falsetti, PhD of Cambridge Biomarketing discusses the role of rare disease advocacy groups.