JULY 7, 2017
The Rare Disease Advisory Council Act, Act 14
The Rare Disease Advisory Council Act, Act 14, was enacted on July 7, 2017 by Pennsylvania Governor Tom Wolf. The Secretary of Health, Rachel L. Levine, M.D, designated Bureau Director Tomas J. Aguilar from the Department of Health as the Chair.
The Pennsylvania Rare Disease Advisory Council (PARDAC) today brought together rare patients, caregivers, advocates, policymakers and health care professionals at its inaugural Patient Stakeholder Summit. The summit included the unveiling of key initial findings of PARDAC’s ongoing Pennsylvania Rare Disease Needs Assessment survey, gathering Pennsylvania-specific insights from rare disease patients and caregivers from across the Commonwealth, as well as plenary sessions focused on the topics of gene therapy and newborn screening.
Dr. David Fajgenbaum of Merion Station, PA is the Co-Founder and President of the Castleman Disease Collaborative Network, a global initiative dedicated to accelerating research and treatment for Castleman disease by facilitating collaboration among the global research community, mobilizing resources, strategically investing in high-impact research, and supporting patients and their loved ones.
Dr. Nicole Henwood of West Chester, PA is the founder, President and CEO of NF2 BioSolutions which is seeking a cure or preventative treatment for neurofibromatosis type 2 by vigorously supporting and advancing existing and next-generation biomedical technologies, such as gene therapy and immunotherapy.
Leah Schust Myers of Gettysburg, PA is the Founder, Executive Director and Chair of Research at FamilieSCN2A Foundation which aims to improve the lives of those affected by SCN2A related disorders through research, public awareness, family support and patient advocacy.
Joe Coyne of Gilbertsville, PA is the Founder and Executive Director of Garrett the Grand – Batten Fighter which focuses on creating awareness, funding trials and research for Batten Disease, helping future health care providers with a scholarship and providing opportunities through a wellness running program.
Lisa Butler of Blue Bell, PA is the Executive Director of the GBS|CIDP Foundation International which aims to improve the quality of life for individuals and families affected by Guillain Barre Syndrome, Chronic Inflammatory Demyelinating Polyneuropathy, and related conditions through support, education, research and advocacy.
Emily Kramer-Golinkoff of Ardmore, PA is the co-founder of Emily’s Entourage which accelerates research and drug development for nonsense mutations of cystic fibrosis.
Rob Long of Chester Springs, PA is the Executive Director of Uplifting Athletes Inc. which inspires the rare disease community with hope through the power of sport.
Aviva Rosenberg of Pittsburgh, PA is the Co-Founder and Co-President of the Gaucher Community Alliance which supports patients with Gaucher disease and their families through peer-to-peer support and education, advocacy, patient and family resources, and networking.
Caryl Harris of New Hope, PA is the Executive Director of Avery’s Hope which raises money and awareness for rare pediatric GI patient families assisting with out-of-pocket and insurance denied expenses.