AdvocacyLeading advocates and experts share their experiences
- Jen McNary Discusses Rare Disease Week and the Role of Advocates
Jennifer McNary is a consultant, speaker, and rare disease advocate. She is also a mother of two boys with Duchenne muscular dystrophy. In this video, Jennifer discusses Rare Disease Day and her evolving role of Advocate to Industry consultant.
- NIH Director Francis Collins Talks About Rare Disease Day
Dr. Collins began his talk by acknowledging that Rare Diseases are more relevant that ever with all of NIH’s 27 institutes and centers having some investment in rare disease research.
- Kyle Bryant Discusses Friedreich’s Ataxia and Rare Disease Day
Kyle Bryant is a rare disease advocate. In this video, Kyle discusses Friedreich’s Ataxia (the rare disease he lives with) and Rare Disease Day. Kyle was diagnosed with Friedreich’s Ataxia at the age of 17.
- Rare Disease Day 2019
Rare Disease Day is a day for those in the rare disease community to share their stories. There are over 7000 rare diseases and each person who has a rare disease, or who is working to find a treatment for a rare disease, has a unique story to tell. Ch …
- Rare Disease Week Legislative Conference and Lobbying Efforts
Steve Silvestri, Director of Public Policy at the EveryLife Foundation for Rare Diseases, discusses the role of advocates in lobbying Congress and the 2019 Legislative Conference Agenda held February 25, 2019 at the Ronald Reagan Building and International Trade Center in Washington, DC.
- 2019 BIO International Convention
James C. Greenwood, President and CEO of the Biotechnology Innovation Organization (BIO) discusses the upcoming 2019 BIO International Conference to be held June 3-6 in Philadelphia, PA.
- Leaving No One Behind: Rare Disease Day at the United Nations
The United Nations’ non-governmental organization (NGO) Committee for Rare Disease held its Rare Disease Day event on February 21, 2019 at the United Nations Headquarters in New York City. The event included a who’s who of the international rare diseas …
- Rare Disease Day: Spotlight on Rare Disorders and the Need for Clinical Trials
February 28 is the 12th Annual Rare Disease Day, the theme of which this year is “bridging health and social care”. This day brings together the rare disease community and highlights the need for better coordination of medical, social, and support services to lessen the burden that rare diseases have on the daily lives of patients, their families, and caregivers.
- 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).
- Living 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 …