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22nd Orphan Drugs and Rare Diseases Global Congress 2024 Americas

Addressing Scientific and Regulatory Challenges to Advance New Treatments for Rare Diseases

Thursday 14th - Friday 15th November 2024


Rogerio Vivaldi

President, CEO

Sigilon Therapeutics, an Eli Lilly and Company

Caroline Kurtz

Chief Development Officer


Siddhart Jain

Medical Director, Global Medical Lead


Andrea C. Furia-Helms, M.P.H.

Director, Patient Affairs Staff, Office of Clinical Policy and Programs, Office of the Commissioner, U.S. Food and Drug Administration

U.S. Food and Drug Administration

Christine Von Raesfeld

Founder & CEO, People with Empathy

People with Empathy

Nigel Nicholls

General Manager Northern European Cluster

Egetis Therapeutics AB

Tricha Shivas, MBe

Chief Strategy Officer

Foundation for Sarcoidosis Research

Joslyn Crowe, MSW, MA

Vice President

International Niemann-Pick Disease Alliance (INPDA)

Monica L Weldon

President/CEO/Founder Bridge the Gap - SYNGAP Education and Research Foundation

Bridge the Gap - SYNGAP Education and Research Foundation

Giacomo Chiesi

Head of Chiesi Global Rare Diseases

Chiesi Group

Stuart Siedman

Head of Patient Advocacy

Chiesi Global Rare Diseases

Craig Martin

CEO, Global Genes

Global Genes

Frank Rivera

Co-Founder/President- Stronger Than Sarcoidosis

Stronger Than Sarcoidosis

Sanjeev Luther

President, CEO & Chairman of the Board, Cornerstone Pharmaceuticals

Cornerstone Pharmaceuticals

Michael Eging

Founder, Executive Director

Rare Access Action Project (RAAP)

Jayson Slotnik

Principal and Founding Partner

Health Policy Strategies

Shane Hegarty

Chief Scientific Officer & Co-Founder

AXONIS Therapeutics, Inc.

Tara J. Britt

Founder and President, Rare Disease Innovations Institute, Inc.

Rare Disease Innovations Institute, Inc.

Julie Yu

Chief Program Officer

Amicus Therapeutics

David Young

President, Research & Development

Processa Pharmaceuticals, Inc.

Jennifer McNary

Exec. Director, Head, Patient Advocacy & Engagement, Fulcrum Therapeutics

Fulcrum Therapeutics

Annette Maughan


KBG Foundation

Monique Mulkern

Head of Global Launch Excellence

Alexion, AstraZeneca Rare Disease

Mohamed H. Ladha

President & GM, North America


Jeremy Griffin

Executive Director, New York City Hemophilia Chapter

New York City Hemophilia Chapter

Andrew Leger

Head of US Business Development


Shane Hegarty

Chief Scientific Officer & Co-Founder, AXONIS Therapeutics, Inc

AXONIS Therapeutics, Inc

Patricia Weltin

CEO and Founder of the Beyond the Diagnosis

Beyond the Diagnosis

Samuel L. Seward, Jr. MD

Professor and Chair, Department of Medicine

Mount Sinai

Eugean Jiwanmall

Senior Research Analyst for Medical Policy & Technology Evaluation, Independence Blue Cross

Independence Blue Cross

Kristin Smedley

President, Curing Retinal Blindness Foundation

Curing Retinal Blindness Foundation

Smitha Jagadish

Vice President, Head of Rare Diseases and New Modalities


Alison Schecter

Chief Executive Officer

Molecules to Medicine Advisory Group

Emily M. Parks

Founder, CEO


Julie Breneiser

Executive Director

Gorlin Syndrome Alliance

Alan J. Balch

Chief Executive Officer

National Patient Advocate Foundation

Ruth Jacob O’Keefe


ThinkGenetic, Inc.

Ryan Miller

Director of Clinical Science and Diagnostics

PTC Therapeutics

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