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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.
HARRISBURG, PA – The Pennsylvania Rare Disease Advisory Council (PARDAC) hailed the recent passage of Act 133 amends the Newborn Child Testing Act to mandate that 37 disorders be screened at birth. Newborn screening is one of the most critical public health programs in the United States. Before this legislation, in addition to tests for…
HARRISBURG, PA — The Pennsylvania Rare Disease Advisory Council (PARDAC) today called upon the rare disease community to participate in a first-of-its-kind statewide needs assessment. The results of the survey will help provide a better picture of what rare disease patients face and guide the state’s future policies and initiatives related to rare diseases.“Since the…