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
KEY INDUSTRY EXPERT SPEAKERS
- Sanjeev Luther, President & CEO, Cornerstone Pharmaceuticals
- Caroline Kurtz, Chief Development Officer, Synlogic
- Giacomo Chiesi, Head of Global Rare Diseases, Chiesi
- Stuart Siedman, Global Head Patient Advocacy, Rare Diseases, Chiesi
- Nigel Nicholls, General Manager Northern European Cluster, Egetis Therapeutics AB
- Joslyn Crowe, Vice President, International Niemann-Pick Disease Alliance (INPDA)
- Tricha Shivas, Chief Strategy Officer, Foundation for Sarcoidosis Research
- Siddharth Jain, Medical Director –LSDs & HAE, Global Medical Affairs, Takeda
- Amit Rakhit, MD, Chief Executive Officer, Flare Therapeutics
- Mohamed H. Ladha, President & GM, North America, RECORDATI RARE DISEASES
- Rogerio Vivaldi, President, CEO, Sigilon Therapeutics, an Eli Lilly and Company
- David Young, President, Research & Development, Processa Pharmaceuticals, Inc.
- Tara J. Britt, Founder and President, Rare Disease Innovations Institute, Inc.
- Michael Eging, Founder, Executive Director, Rare Access Action Project (RAAP)
- Kelsey Carreon, Patient Engagement, Rare Access Action Project (RAAP)*
- Kristin Smedley, President, Curing Retinal Blindness Foundation
- Smitha Jagadish, Vice President, Head of Rare Diseases and New Modalities, BIAL
- Monique Mulkern, Head of Global Launch Excellence, Alexion, AstraZeneca Rare Disease
- David Ross, Patient Advocate, Rare Disease Mental Health
- Monica L Weldon, President/CEO/Founder, SYNGAP1 Foundation
- Samuel L. Seward, Jr. MD, Professor and Chair, Department of Medicine, Mount Sinai
- Jennifer McNary, Founder, One Rare
- Frank Rivera, Co-Founder/President, Stronger Than Sarcoidosis
- Andrew Leger, Head of US Business Development, Ferrer
- Craig Martin, President, Chief Engagement Officer, Rithm Health
- Eugean Jiwanmall, Sr Research Analyst, Independence Blue Cross
- Emily M. Parks, Founder, CEO, POP!
- Patricia Weltin, CEO/Founder, Beyond the Diagnosis
- Jeremy Griffin, Executive Director, New York City Hemophilia Chapter
- Senior Representative, CureDuchenne
- Jayson Slotnik, Principal and Founding Partner, Health Policy Strategies
- Shane Hegarty, Chief Scientific Officer & Co-Founder, AXONIS Therapeutics, Inc.
- Julie Yu, Chief Program Officer, Amicus Therapeutics
- Samantha K. Zappia, VP, Regulatory Affairs & Quality, Vigil Neuroscience, Inc.
- Julie Breneiser, Executive Director, Gorlin Syndrome Alliance
- Annette Maughan, CEO/Co-Founder, KBG Foundation
- Jodi M. Wolff, Vice President, Patient Advocacy & Engagement, Rejuvenate Bio
- Alison Schecter, Chief Executive Officer, Molecules to Medicine Advisory Group
- Julie Breneiser, Executive Director, Gorlin Syndrome Alliance
- Alan J. Balch, Chief Executive Officer, National Patient Advocate Foundation
- Ruth Jacob O’Keefe, CEO, ThinkGenetic, Inc.
- Ryan Miller, Director of Clinical Science and Diagnostics, PTC Therapeutics
![](https://orphandrugsus.com/wp-content/uploads/2023/03/R-Vivaldi-1.png)
Rogerio Vivaldi
President, CEO
![](https://orphandrugsus.com/wp-content/uploads/2023/08/C-Kurts.png)
Caroline Kurtz
Chief Development Officer
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Siddhart Jain
Medical Director, Global Medical Lead
![](https://orphandrugsus.com/wp-content/uploads/2023/03/39.png)
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
![](https://orphandrugsus.com/wp-content/uploads/2023/03/39.png)
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
![](https://orphandrugsus.com/wp-content/uploads/2021/09/C-V-Raesfeld-1.png)
Christine Von Raesfeld
Founder & CEO, People with Empathy
![](https://orphandrugsus.com/wp-content/uploads/2023/08/N-Nicholls-1.png)
Nigel Nicholls
General Manager Northern European Cluster
![](https://orphandrugsus.com/wp-content/uploads/2023/08/T-Shivas.png)
Tricha Shivas, MBe
Chief Strategy Officer
![](https://orphandrugsus.com/wp-content/uploads/2023/08/J-Crowe.png)
Joslyn Crowe, MSW, MA
Vice President
![](https://orphandrugsus.com/wp-content/uploads/2023/03/monica.weld_.png)
Monica L Weldon
President/CEO/Founder Bridge the Gap - SYNGAP Education and Research Foundation
Bridge the Gap - SYNGAP Education and Research Foundation
![](https://orphandrugsus.com/wp-content/uploads/2023/03/monica.weld_.png)
Monica L Weldon
President/CEO/Founder Bridge the Gap - SYNGAP Education and Research Foundation
![](https://orphandrugsus.com/wp-content/uploads/2023/08/G-Chiesi.png)
Giacomo Chiesi
Head of Chiesi Global Rare Diseases
![](https://orphandrugsus.com/wp-content/uploads/2023/08/S-Siedman.png)
Stuart Siedman
Head of Patient Advocacy
![](https://orphandrugsus.com/wp-content/uploads/2023/03/C-Martin-1.png)
Craig Martin
CEO, Global Genes
![](https://orphandrugsus.com/wp-content/uploads/2023/08/F-Rivera-2.png)
Frank Rivera
Co-Founder/President- Stronger Than Sarcoidosis
![](https://orphandrugsus.com/wp-content/uploads/2023/09/S-Luther.png)
Sanjeev Luther
President, CEO & Chairman of the Board, Cornerstone Pharmaceuticals
Cornerstone Pharmaceuticals
![](https://orphandrugsus.com/wp-content/uploads/2023/09/S-Luther.png)
Sanjeev Luther
President, CEO & Chairman of the Board, Cornerstone Pharmaceuticals
![](https://orphandrugsus.com/wp-content/uploads/2023/08/M-Eging.png)
Michael Eging
Founder, Executive Director
![](https://orphandrugsus.com/wp-content/uploads/2023/08/J-Slotnik.png)
Jayson Slotnik
Principal and Founding Partner
![](https://orphandrugsus.com/wp-content/uploads/2023/08/Hegarty.png)
Shane Hegarty
Chief Scientific Officer & Co-Founder
![](https://orphandrugsus.com/wp-content/uploads/2023/03/T-Britt.png)
Tara J. Britt
Founder and President, Rare Disease Innovations Institute, Inc.
Rare Disease Innovations Institute, Inc.
![](https://orphandrugsus.com/wp-content/uploads/2023/03/T-Britt.png)
Tara J. Britt
Founder and President, Rare Disease Innovations Institute, Inc.
![](https://orphandrugsus.com/wp-content/uploads/2023/08/Julie-Yu.png)
Julie Yu
Chief Program Officer
![](https://orphandrugsus.com/wp-content/uploads/2023/08/D-Young.png)
David Young
President, Research & Development
![](https://orphandrugsus.com/wp-content/uploads/2023/03/McNary-J.png)
Jennifer McNary
Exec. Director, Head, Patient Advocacy & Engagement, Fulcrum Therapeutics
Fulcrum Therapeutics
![](https://orphandrugsus.com/wp-content/uploads/2023/03/McNary-J.png)
Jennifer McNary
Exec. Director, Head, Patient Advocacy & Engagement, Fulcrum Therapeutics
![](https://orphandrugsus.com/wp-content/uploads/2023/08/A-Maughan.png)
Annette Maughan
CEO/Co-Founder
![](https://orphandrugsus.com/wp-content/uploads/2023/09/Monique-1.png)
Monique Mulkern
Head of Global Launch Excellence
![](https://orphandrugsus.com/wp-content/uploads/2023/03/J-Griffin.png)
Jeremy Griffin
Executive Director, New York City Hemophilia Chapter
New York City Hemophilia Chapter
![](https://orphandrugsus.com/wp-content/uploads/2023/03/J-Griffin.png)
Jeremy Griffin
Executive Director, New York City Hemophilia Chapter
![](https://orphandrugsus.com/wp-content/uploads/2023/09/A-leger.png)
Andrew Leger
Head of US Business Development
![](https://orphandrugsus.com/wp-content/uploads/2023/03/Hegarty-S.png)
Shane Hegarty
Chief Scientific Officer & Co-Founder, AXONIS Therapeutics, Inc
AXONIS Therapeutics, Inc
![](https://orphandrugsus.com/wp-content/uploads/2023/03/Hegarty-S.png)
Shane Hegarty
Chief Scientific Officer & Co-Founder, AXONIS Therapeutics, Inc
![](https://orphandrugsus.com/wp-content/uploads/2023/03/Web-Image-39.png)
Patricia Weltin
CEO and Founder of the Beyond the Diagnosis
![](https://orphandrugsus.com/wp-content/uploads/2023/09/S-Seward.png)
Samuel L. Seward, Jr. MD
Professor and Chair, Department of Medicine
![](https://orphandrugsus.com/wp-content/uploads/2023/03/Eugean-3.png)
Eugean Jiwanmall
Senior Research Analyst for Medical Policy & Technology Evaluation, Independence Blue Cross
Independence Blue Cross
![](https://orphandrugsus.com/wp-content/uploads/2023/03/Eugean-3.png)
Eugean Jiwanmall
Senior Research Analyst for Medical Policy & Technology Evaluation, Independence Blue Cross
![](https://orphandrugsus.com/wp-content/uploads/2023/08/Smedley-1.png)
Kristin Smedley
President, Curing Retinal Blindness Foundation
![](https://orphandrugsus.com/wp-content/uploads/2023/09/Smitha.png)
Smitha Jagadish
Vice President, Head of Rare Diseases and New Modalities
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Alison Schecter
Chief Executive Officer
![](https://orphandrugsus.com/wp-content/uploads/2023/10/E-Parks-1.png)
Emily M. Parks
Founder, CEO
![](https://orphandrugsus.com/wp-content/uploads/2023/10/J-Breneiser.png)
Julie Breneiser
Executive Director
![](https://orphandrugsus.com/wp-content/uploads/2023/10/Balch-A.png)
Alan J. Balch
Chief Executive Officer
![](https://orphandrugsus.com/wp-content/uploads/2023/10/R-OKeefe-1.png)
Ruth Jacob O’Keefe
CEO
![](https://orphandrugsus.com/wp-content/uploads/2021/12/Web-Image-10.png)
Available Shortly
Coming Soon!
![](https://orphandrugsus.com/wp-content/uploads/2021/12/Web-Image-10.png)
Available Shortly
![](https://orphandrugsus.com/wp-content/uploads/2021/12/Web-Image-13-1.png)
Available Shortly
Coming Soon!
![](https://orphandrugsus.com/wp-content/uploads/2021/12/Web-Image-13-1.png)