The Pennsylvania Rare Disease Day event is to empower rare disease patients to effectively share their stories and gain the knowledge around the legislative process to influence change. We have the additional goal of educating and informing our legislators on issues affecting rare disease patients, and how they too can most effectively work with them to provide better outcomes throughout the Commonwealth.
Rare disease patients throughout Pennsylvania are faced with unique, often life-threatening challenges each and every day. Of the nearly 10,000 rare diseases in the US, only a few hundred have any established treatment. Life Sciences PA, in partnership with the National Organization for Rare Disorders (NORD) and the Pennsylvania Rare Disease Advisory Council (PARDAC), invite you to join us in this very important discussion as we understand the challenges facing the rare disease community throughout the Commonwealth, discuss the path forward, and work together to create the innovative ecosystem that rare disease patients depend on.
Agenda:
8:30 a.m. – 8:40 a.m. – Welcome by Life Sciences PA President and CEO, Christopher P. Molineaux
8:45 a.m. – 9:25 a.m. - Panel Discussion around the Orphan Drug Tax Credit and proposed changes
- Dane Christiansen, Managing Partner, Health and Medicine Counsel
- Heidi Ross, Acting Vice President of Policy & Regulatory Affairs, NORD
- Geoff Werth, Head of Government Relations, Harmony Biosciences
9:30 a.m. – 10:00 a.m. – Update on The Bespoke Gene Therapy Consortium (BGTC)
- Courtney Silverthorn, Associate Vice President, Research Partnerships, Foundation for the National Institutes of Health (FNIH)
10:05 a.m. – 10:45 a.m. – Impact of Newborn Screening Bill in PA
- Lesa Brackbill, Outreach Coordinator for Policy and Advocacy, Leukodystrophy Newborn Screening Action Network
- Senator John DiSanto, 15th Senatorial District
- Meagan Perry, Director of Public Affairs & Advocacy, Orchard Therapeutics
10:50 a.m. – 11:00 a.m. – Update from PARDAC, Invitation to Press Conference
- Stephanie Fischer, Patient Advocate and Council Member, Pennsylvania Rare Disease Advisory Council
If you have any questions about the event, please contact Nicole Oshurak, Vice President, State Policy & Public Affairs, [email protected]
