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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.

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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.

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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.

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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.

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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.

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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.

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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…

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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…

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HARRISBURG, Pa. – An estimated 1.2 million Pennsylvanians are diagnosed with a rare disease, often living in isolation with significant challenges in obtaining medical treatment and insurance to cover their care. On Wednesday, September 25, some of these brave Pennsylvanians traveled to the State Capitol and shared their personal stories of struggle and achievement while…

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HARRISBURG – In honor of National Rare Disease Day on Feb. 28, Reps. Marcy Toepel (R-Montgomery) and Mark Longietti (D-Mercer), as well as Sens. Judy Ward (R-Blair/Cumberland/Franklin/Fulton/Huntingdon) and John Blake (D-Lackawanna/Luzerne/Monroe) will serve as co-chairs for Pennsylvania’s Rare Disease Caucus for the 2019-20 Legislative Session.  News headings Toepel, Longietti, Ward and Blake will work with…

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