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Agenda at a Glance

Note: titles and times subject to change
Day 1 Evening Session Wednesday, October 29, 2025
Introduction & Greetings from PRF
Medical Director Leslie Gordon, MD, PhD, Executive Director Audrey Gordon, Esq., Board Chair Scott Berns, MD, MPH, FAAP
Merlin Waldron (moderator), Megan Nighbor, Griffin Ray, Ricardo Zanolli, Nathan Falcone, Michiel Vandeweert “Coming of Age” Conversations:  Young adults with HGPS as Research Partners

Musical Kick-off with Francis Collins, MD, PhD National Human Genome Research Institute, National Institutes of Health, Bethesda, MD

DINNER and Networking Evening
Day 2 Morning Session Thursday, October 30, 2025

Current Treatment Trial Outcomes in Progeria                               Moderator: Mark Kieran, MD, PhD, Day One Bio.
Dr. Leslie Gordon, PhD
Brown University, Hasbro Children’s Hospital, Boston		 Long-term Natural History Lonafarnib Treatment Trial
Dra. Mônica Kleinman
Boston Children’s Hospital, Boston, MA		 Phase 2a Progerinin Clinical Treatment Trial for Progeria
Barbara Natke, PhD
Progeria Research Foundation, Peabody, MA		 Tools for Pivotal Science and Medicine in Progeria:
Antibody and Biomarker Assay Development
Sunil Ghelani, MD
Progeria Research Foundation, Peabody, MA		 First-ever Aortic and Coronary Artery Calcium Computed Tomography Clinical Trial Study in Progeria

LUNCH, Informal Poster Viewings and Networking 
Day 2 Afternoon Session Thursday, October 30, 2025

Cardiovascular Disease in HGPS and Aging                                        Moderator: Maria Eriksson, PhD
Maria Eriksson, PhD
Karolinska Institute, Sweden
 Single-cell analysis reveals the phenotypic landscape of arterial cells in Progeria

Shannon Lyon, MD
Boston Children’s Hospital, Boston, MA

Keri Shafer, MD
UT Southwestern Medical Center, Fort Worth, Texas

Sheila Hegde, MD
Brigham & Women’s Hospital, Boston, MA

 New discoveries in Cardiac Measures of Disease in Progeria and Aging through Echocardiography
Kan Cao, PhD
Univ. of Maryland, College Park, MD
 Angiopoietin-2 reverses endothelial cell dysfunction in Progeria vasculature
Ricardo Villa Bellosta, PhD
CiMUS and USC, Av. Barcelona, Spain		 Progeria and Vascular Calcification: Diet and Treatments
LATE-BREAKING DATA PRESENTATION To Be Determined – Cutting Edge Discoveries
    7th INNING STRETCH and Networking Time
Sessão de pôsteres relâmpago 1 minuto e 1 slide por apresentador de pôster
DINNER and Informal Conversation
Day 2 Evening Poster Session Thursday, October 30, 2025

 7:00-8:00 Even-Numbered Posters Formally Presented                           8:00-9:00 Odd-Numbered Posters Formally Presented
Day 3 Sunrise Session: Coffee Talk with Patients and Families Friday, October 31, 2025
Day 3 Morning Session Friday, October 31, 2025

Progeria Treatment Discovery for a Healthy Future                               Moderator: Francis Collins, MD, PhD
David Liu, PhD, Broad Institute, MIT, Cambridge, MA &
Dr. Francis Collins, PhD		  ProSPER: PROgeria Streamlined Platform gene Editing for Rare diseases
Nizar Saad, PhD
Nationwide Children’s Hospital, Columbus, OH		 Development of a miRNA-based Gene Therapy for Hutchinson-Gilford Progeria Syndrome (HGPS)
Ning Shen, PhD, Professor
Zhejiang Univ. Medical School, Hangzhou, China		 AI-driven therapeutic antisense oligonucleotide for Hutchinson-Gilford Progeria
Silvia Ortega Gutierrez, PhD
Complutense University, Madrid Spain		 Reduction of progerin levels by small molecules as a new approach for treating Progeria

LUNCH and Networking 
Day 3 Afternoon Session Friday, October 31, 2025

Endocrine and GI Disease in Progeria                                                  Moderator:  Vicente Andres, PhD                  
Vicente Andrés, PhD
Spanish National CV Res. Center, Madrid, Spain		 Lipodystrophy in Progeria and Aging
Laurence Arbibe, PhD
INSERM Institut Necker Enfants Malades, Paris, Fr.		 Unraveling accelerated intestinal ageing in HGPS physiopathology: an integrative approach
Susana Gonzalo, PhD
St. Louis University, St Louis, MO		 Cathepsin-L mediated remodeling of the nuclear proteome
LATE BREAKING DATA PRESENTATION To Be Determined – Cutting Edge Discoveries
Poster Elevation Talk by Junior Investigator To Be Determined after Poster Abstract Submissions

NETWORKING TIME
Workshop Wrap-up:
All-hands Meeting Discussion on Key Findings and Highest Priorities for Future Success

Moderators:  Francis Collins, Mark Kieran, Leslie Gordon

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