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Innovation in Rare Disease: Making Progress with Cell & Gene Therapies
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Jump to the Agenda for Each Day: Day One | Day Two | Day Three
This Rare Disease Day, Syneos Health continues the drive to raise awareness of rare diseases and their impact on patients’ lives – and to shine a light on the opportunities for cell and gene therapies in treating these disorders.
Join us for an inspiring program that brings together – over three days – patients and their families, legislators, industry experts, advocates and sponsors for presentations and panel discussions on education initiatives, recent advancements and the future promise of cell and gene medicine, and current patient experiences with these therapies.
Day One | February 23rd, 2021 | 9am - 10:15am ET
All times are listed in ET
9:00am | Welcome & Opening Remarks
Dr. Raymond Huml, Vice President and Head of the Rare Disease Consortium, Syneos Health
Tara J. Britt, Founder and President, Rare Disease Innovations Institute, Associate Chair, North Carolina Rare Disease Advisory Council
9:10am | Keynote Address
Congressman G. K. Butterfield, North Carolina’s First Congressional District and Congressional Co-Chair, Rare Disease Caucus.
9:20am | Overview of Cell & Gene Therapies
Pete Robinson, Vice President and Head of the Cell & Gene Therapy Consortium, Syneos Health
9:35am | Patient Perspectives & Discussion
Hear from industry and family advocates from the Spinal Muscular Atrophy (SMA) community on their experience with gene therapies used to treat the disease.
Introduction by Meredith Huml, Communications Assistant, Disability Advocates of North Carolina
The Pantier Family, SMA Advocates
9:55am | Closing Remarks, Wrap up Day One & Day Two Introduction
Dr. Raymond Huml, Vice President and Head of the Rare Disease Consortium, Syneos Health
Tara Britt, Founder and President, Rare Disease Innovations Institute and Associate Chair, North Carolina Rare Disease Advisory Council
10:00 - 10:15am | Q & A
Open for Q & A and networking with organizers and panelists
Day Two | February 24th, 2021 | 9am - 10:15am ET
9:00am | Welcome & Opening Remarks
Dr. Raymond Huml, Vice President and Head of the Rare Disease Consortium, Syneos Health
Tara Britt, Founder and President, Rare Disease Innovations Institute and Associate Chair, North Carolina Rare Disease Advisory Council
9:05am | STRiDE State Rare Disease Education Forums
Morrie Ruffin, Executive Director, ARM Foundation
Tara Britt, Founder and President, Rare Disease Innovations Institute and Associate Chair, North Carolina Rare Disease Advisory Council
9:20am | Patient Perspectives & Discussion
Hear from patients and patient advocates about their experience with Cell and Gene Medicine and rare disease.
Introduction by Pete Robinson, Vice President and Head of the Cell & Gene Therapy Consortium, Syneos Health
Patient Panelists:
Justina Williams (Sickle Cell)
Jenny Klein (MPS)
Kevin Schaefer (SMA)
Clayton Goldin (Mitochondrial Deficiency)
Charlene Cowell (Hemophilia)
Moderators:
Parvathy Krishnan, MS, RD, CNSC, LDN, Director, Patient Engagement, Rare Disease Innovations Institute
Keri McDonough, Lead, Advocacy and Patient Relations, Syneos Health
9:45am | Closing Remarks, Wrap up Day Two & Day Three Introduction
Dr. Raymond Huml, Vice President and Head of the Rare Disease Consortium, Syneos Health
Pete Robinson, Vice President and Head of the Cell & Gene Therapy Consortium, Syneos Health
Tara Britt, Founder and President, Rare Disease Innovations Institute and Associate Chair, North Carolina Rare Disease Advisory Council
9:50am | Q & A
Open for Q & A and networking with organizers and panelists
Day Three | February 25th, 2021 | 9am - 10:15am ET
9:00am | Welcome & Opening Remarks
Dr. Raymond Huml, Vice President and Head of the Rare Disease Consortium, Syneos Health
9:05am | Disease Specific Advancements in Cell and Gene Medicine
Pete Robinson, Vice President and Head of the Cell & Gene Therapy Consortium, Syneos Health
Dr. Maryna Kolochavina, Executive Director, Syneos One, Syneos Health
9:20am | Panel Discussion
Hear from experts in the field of retinitis pigmentosa (RP) on the current status and future of Cell and Gene Medicine.
Dr. Cadmus Rich, Chief Medical Officer, Aura Biosciences
Parvathy Krishnan, MS, RD, CNSC, LDN, Director, Patient Engagement, Rare Disease Innovations Institute
Dr. Van K. Duesterberg, patient, Director of Automation Engineering, and Founder of Patient Led
Dr. Todd Durham, Vice President, Clinical & Outcomes Research, Foundation for Fighting Blindness
9:50am | Closing Remarks for the Workshop
Dr. Nick Kenny, Chief Scientific Officer, Syneos Health
Tara Britt, Founder and President, Rare Disease Innovations Institute and Associate Chair, North Carolina Rare Disease Advisory Council
10:00 - 10:15am | Q & A
Open for Q & A and networking with organizers and panelists
When
Tue, Feb 23, 09:00 am to Thu, Feb 25, 10:15 am
Additional Details
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