Newborn Screening: Toward a System That Keeps Pace with Gene Therapy Advances
Register separately for one or more workshops on May 15

Newborn Screening: Toward a System That Keeps Pace with Gene Therapy Advances

All times below listed in ET

Planned by the Government Relations Committee.
Cochairs: Meagan Perry and Diane Berry, PhD

Register for Newborn Screening: Toward a System That Keeps Pace with Gene Therapy Advances

Session 1 – The Recommended Uniform Screening Panel (RUSP) Current and Future Process

8:00 – 8:55 a.m.

Potential Solutions to Prepare Newborn Screening for Transformative Therapies
Don Bailey, PhD, RTI International

Streamlining Future RUSP Applications
Terri Klein, National MPS Society

Roundtable Panel: The RUSP Current and Future Process
Don Bailey, PhD, RTI International
Terri Klein, National MPS Society
Elisa Seeger, ALD Alliance

Moderated by Kent Christopherson, Orchard Therapeutics and Stacey Frisk, Sarepta

Session 2 – State Implementation and Innovation

8:55 – 9:45 a.m.

Hastening the State Uptake of Newly Approved Conditions
Erin Frey, Ultragenyx

What Can States Do Before a Condition is Added to the RUSP?
Julie Morrison, Illinois State Senate

Roundtable Panel: State Implementation and Innovation
Erin Frey, Ultragenyx
Annie Kennedy, EveryLife Foundation
Julie Morrison, Illinois State Senate
Scott Shone, PhD, North Carolina State Laboratory of Public Health

Moderated by Kent Christopherson, Orchard Therapeutics and Stacey Frisk, Sarepta

Break: 9:45 – 10:15 a.m.

Session 3 – Data Collection and Newborn Screening Programs

10:15 – 11:05 a.m.

Data Collection and Newborn Screening Programs: Opportunities of a Multi-Disease Panel Approach
Melissa Wasserstein, MD, Montefiore Medical Center

Data Collection and Newborn Screening Programs: State and Regional Pilot Programs
Joseph Orsini, PhD, New York Department of Health

Roundtable Panel: Data Collection and Newborn Screening Program
Melissa Wasserstein, MD, Montefiore Medical Center
Joseph Orsini, PhD, New York Department of Health
Barbara Burton, MD, Lurie’s Children’s Hospital
Amy Brower, PhD, American College of Genetics and Genomics

Moderated by Kent Christopherson, PhD, Orchard Therapeutics and Stacey Frisk, Sarepta

Session 4 – Evaluating Emerging Screening Methods and Clinical Programs

11:05 a.m. – 12:00 p.m.

Evaluating Emerging Screening Methods and Clinical Programs
Stephen Kingsmore, MD, DSc, Rady Children’s Hospital

Evaluating Emerging Screening Methods and Clinical Programs
Richard Parad, MD, Brigham and Women’s Hospital

Roundtable Panel: Evaluating Emerging Screening Methods and Clinical Programs
Stephen Kingsmore, MD, DSc, Rady Children’s Hospital
Richard Parad, MD, Brigham and Women’s Hospital
Phil Reilly, MD, JD, Third Rock Ventures

Moderated by Kent Christopherson, Orchard Therapeutics and Stacey Frisk, Sarepta

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