January 29 – 30, 2024 Meeting

Agenda (PDF - 212 KB)

Meeting Minutes - Pending

Webcast

• January 29, 2024 
• January 30, 2024 

Transcripts

• January 29, 2024 (PDF - 672 KB)
• January 30, 2024 (PDF - 429 KB)

Presentations

Day 1

Family Outcomes of Newborn Screening: Project Overview Update (PDF - 1 MB)

Don Bailey, Jr., Ph.D., Med RTI International

Panel: Research on Family Perspectives:

Families’ Search for Meaning and Value in Rare Genetic Diagnoses (PDF - 1 MB)
Sara Ackerman, Ph.D., M.Ed.
University of California, San Francisco

The “Value of Values”: Expanding Assessment of Net Benefits and Harms through Social Science Data (PDF - 1 MB)
Aaron Goldenberg, Ph.D., M.A., M.P.H.
Case Western Reserve University School of Medicine

Duchenne Muscular Dystrophy Evidence-Based Review: Phase 2 Update (Presentation Forthcoming)

Alex R. Kemper, M.D., M.P.H., M.S.
Lead Evidence-Based Review Group

ACHDNC Decision Matrix Tool: Public Health Assessment (PDF - 163 KB)

Ned Calonge, M.D., M.P.H.
Committee Chair

ACHDNC Nomination Process Update (PDF - 298 KB)

Ned Calonge, M.D., M.P.H.
Committee Chair

ACHDNC Nomination and Evidence-Based Review Process Proposal (PDF - 226 KB)

Ned Calonge, M.D., M.P.H.
Committee Chair

Day 2

Newborn Screening for Krabbe Disease Expedited Evidence-Based Review (PDF - 985 KB)

Alex R. Kemper, M.D., M.P.H., M.S.
Lead, Evidence-Based Review Group

Lisa A. Prosser, Ph.D.
Member, Evidence-Based Review Group

Committee Report: Newborn Screening for Krabbe Disease (PDF - 948 KB)

Shawn E. McCandless, M.D.
Committee Member

Jennifer M. Kwon, M.D., M.P.H., FAAN
Committee Member

*Note: While the presenters did not recommend Krabbe disease for inclusion to the RUSP, the Committee ultimately voted to recommend to the Secretary to add Krabbe disease for inclusion to the RUSP. (2/5/2024)