{"database": "press", "table": "releases", "rows": [["https://www.paul.senate.gov/news-icymi-dr-rand-paul-holds-hearing-expediting-treatments-patients-rare-diseases/", "ICYMI: Dr. Rand Paul Holds Hearing on Expediting Treatments for Patients with Rare Diseases", "2018-10-04", "2018", "2018-10", "Republican", "Senate", "KY", "Rand Paul", "P000603", "www.paul.senate.gov", "paul", "https://www.paul.senate.gov/news/", "scraper", "FOR IMMEDIATE RELEASE:\n\nOctober 4, 2018\n\nContact: Press@paul.senate.gov, 202-224-4343\n\nWASHINGTON, D.C. \u2013 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, \u201cRare Diseases: Expediting Treatment for Patients.\u201d\n\nDr. Paul\u2019s 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.\n\n\u201cAs 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,\u201d Dr. Paul remarked in his opening statement.\n\nYou can watch Dr. Paul\u2019s opening statement HERE and the entire hearing HERE.\n\nYou can find a complete list of the witnesses who testified below:\n\nWITNESSES\n\nMark Dant (Louisville, KY)\n\nBoard chair of EveryLife Foundation for Rare Diseases\n\nVolunteer Executive Director, The Ryan Foundation\n\nMark 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.\n\nMarc Patterson, MD\n\nProfessor of Neurology, Pediatrics, and Medical Genetics, Mayo Clinic\n\nDr. Patterson is the chair of the Division of Child and Adolescent Neurology at the Mayo Clinic, where he focuses on rare diseases in children.\n\nMichael Strupp, MD\n\nProfessor at the University of Munich, Germany, in the Department of Neurology and German Center for Vertigo and Balance Disorders\n\nDr. Strupp\u2019s 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.\n\nLincoln Tsang\n\nPartner at Arnold & Porter Kaye Scholer LLP \u2013 Former Head of Biologicals/Biotechnology for the United Kingdom\u2019s Medicines and Healthcare products Regulatory Agency\n\nAt 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.\n\nMallory Factor\n\nFounder, Chairman, and CEO of IntraBio\n\nIntraBio, 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.\n\n###", 1, "2026-03-30T01:40:41Z", "2026-06-27T23:08:35Z"]], "columns": ["url", "title", "date", "year", "month", "party", "chamber", "state", "member_name", "bioguide_id", "domain", "scraper", "source", "date_source", "text", "has_text", "collected_at", "updated_at"], "primary_keys": ["url"], "primary_key_values": ["https://www.paul.senate.gov/news-icymi-dr-rand-paul-holds-hearing-expediting-treatments-patients-rare-diseases/"], "units": {}, "query_ms": 0.9089747909456491, "source": "dwillis/congress-press", "source_url": "https://github.com/dwillis/congress-press", "license": "MIT", "license_url": "https://github.com/dwillis/congress-press/blob/main/LICENSE"}