FOR IMMEDIATE RELEASE:
October 4, 2018
Contact: Press@paul.senate.gov, 202-224-4343
WASHINGTON, D.C. – Yesterday, U.S. Senator Rand Paul (R-KY), chairman of the Children and Families Subcommittee for the Senate Health, Education, Labor and Pensions (HELP) Committee, held a hearing entitled, “Rare Diseases: Expediting Treatment for Patients.”
Dr. Paul’s subcommittee heard from patient advocates, clinicians, and industry professionals as it examined what Congress can do to improve a slow-moving regulatory process that leaves the needs of many patients with rare genetic diseases unmet, as well as how to help ensure these patients have timely access to treatment options to alleviate their conditions.
“As a parent, I can only imagine, though, the overwhelming desperation and hopelessness of being confronted with a fatal diagnosis and being told to simply enjoy the remaining time that their child has left. To the extent that government can improve efficiencies to speed access to treatments for patients and families facing such a situation, such action should be taken without delay,” Dr. Paul remarked in his opening statement.
You can watch Dr. Paul’s opening statement HERE and the entire hearing HERE.
You can find a complete list of the witnesses who testified below:
Mark Dant (Louisville, KY)
Board chair of EveryLife Foundation for Rare Diseases
Volunteer Executive Director, The Ryan Foundation
Mark founded The Ryan Foundation, named for his son who was diagnosed with Mucopolysaccharidosis 1 (MPS I) at age 3 with a life expectancy of 10 years old, and Mark has spent over 25 years working on behalf of the rare disease community. His son, Ryan, graduated from the University of Louisville in 2017.
Marc Patterson, MD
Professor of Neurology, Pediatrics, and Medical Genetics, Mayo Clinic
Dr. Patterson is the chair of the Division of Child and Adolescent Neurology at the Mayo Clinic, where he focuses on rare diseases in children.
Michael Strupp, MD
Professor at the University of Munich, Germany, in the Department of Neurology and German Center for Vertigo and Balance Disorders
Dr. Strupp’s work in drug development has centered around finding known compounds that have a demonstrated safety profile and testing their efficacy for rare and incurable diseases.
Partner at Arnold & Porter Kaye Scholer LLP – Former Head of Biologicals/Biotechnology for the United Kingdom’s Medicines and Healthcare products Regulatory Agency
At Arnold and Porter, Dr. Tsang continues to advise large and small companies working to develop novel technologies to treat pediatric and orphan diseases, and he brings a unique perspective of both the U.S. and EU regulatory systems.
Founder, Chairman, and CEO of IntraBio
IntraBio, founded in 2015, translates research in the fields of lysosomal biology, autophagy, and neurology into orphan drugs and treatments to improve the lives of patients.