2023 RARE Advocacy Summit

September 19, 2023 – September 21, 2023

Register now Watch Live Stream

Connect. Inspire. Learn

Each year, Global Genes convenes one of the world’s largest gatherings of rare disease patients, caregivers, advocates, healthcare professionals, researchers, partners, and allies at the RARE Advocacy Summit.

This is an unparalleled opportunity to forge meaningful connections with others in the rare disease community for future collaboration. Sessions provide attendees with insights about the latest in rare disease innovations, best practices for advocating on an individual and organizational level, and actionable strategies they can take home and implement immediately to accelerate change. This year, the Summit will be held in San Diego California. Join us for networking, learning and inspiration.

Who Should Attend?

* Patient advocacy group leaders

* Rare disease patients

* Caregivers

* Rare disease advocates

2023 Speakers

More speakers for the 2023 RARE Advocacy Summit will be added, so check back for the newest additions!

Kim Aldinger

Seattle Children’s Hospital

Brian Altounian

Just Two Dads Podcast

Carole Bakhos

Jordan’s Guardian Angels

Wendy Benson

Rady Children’s Hospital

Alexandre Bétourné


Ravi Bhargava, MD


Erin Bradshaw

Patient Advocate Foundation

Lea Ann Browning-McNee, MS

Reagan-Udall Foundation

Alex Burgin, PhD

Broad Institute of MIT and Harvard

Jamie Korin Capal

UNC Chapel Hill, Carolina Institute for Developmental Disabilities (CIDD)

Caroline Cheung-Yiu

Community of Undiagnosed Rare and Extraordinary (CURE)

Maya Chopra, MBBS, FRACP

Harvard Medical School

Ryan Colburn

odimm inc

Sati Cooper-McCann

EveryLife Foundation

Chandler Crews

The Chandler Project

Lindsey Cundiff

EveryLife Foundation

Tom D’Amato

Horizon Therapeutics

Mark Dant

Ryan Foundation for Rare Disease Research

Kendall Davis

ICON plc

Michelle Davis

International FOP Association

Daniel DeFabio

Global Genes

Maria Della Rocca, MS, PMP

Global Genes

Scott Demarest, MD

Children’s Hospital Colorado

Aditi Desai, MPH, CPH


Yssa DeWoody, PhD

Ring14 USA

Kira Dies

Rosamund Stone Zander Translational Neuroscience Center, BCH

Suzanne Edison

CURE JM Foundation

Patti Engel

Engage Health

Wendy Erler

Alexion Pharmaceuticals

Lisa Facciolla

US Hereditary Angioedema Association

Nasha Fitter


Matt Flesch

Horizon Therapeutics

Susannah Fox

Author and Digital Health Expert

Albert Freedman, PhD

Freedman Consulting

Simon Frost

Tiber Capital Group

Matthew Fuller


Ron Garber

The Yaya Foundation for 4H Leukodystrophy 

Nicole Glenn, MD


Mike Graglia

Syngap Research Fund

Nathan Grant

Project Alive

Tiara Green

Accessia Health

Lisa Zola Greer

Philanthropy 451

Rachel Groth, PhD


Alaa Hamed, MD, MPH, MBA


Chris Hart, PhD

Creyon Bio

Maureen Hart

Creyon Bio

Jenna Heilman

Hungtington’s Disease Youth Organization

Michele Herndon

Undiagnosed Diseases Network

Melissa Hioco

STXBP1 Foundation

Tom Hume

Cure JM Foundation

Adam Johnson

Rare Disease DadVocate

Arik Johnson, PsyD


Lisa Kadyk, PhD


Jeffrey Kaufman

Adenoid Cystic Carcinoma Research Foundation

Walt Kowtoniuk, PhD

Third Rock Ventures

Parvathy Krishnan

Krishnan Family Foundation

Paul Kruszka, MD, MPH, FACMG


Neil Kumar, MS, PhD

BridgeBio Pharma

Daniel Levine

Levine Media Group

Rob Long

Uplifting Athletes

Jeanette McCarthy, MPH, PhD

Precision Medicine Advisors

Kristin McKay

Project Alive

Kelly McVearry

Bento Biology Platforms

Amy Dockser Marcus

Wall Street Journal

Sarah Marshall, MSW

Undiagnosed Diseases Network

Katherine Maynard


Bojana Mirosaviljevic

ICON plc

Mary Morlino

Global Genes

James O’Brien

International Prader-Wili Syndrome Organization (IPWSO)

Effie Parks

Global Genes

Brian Pfister, PhD, MBA

Praxis Precision Medicines

Mike Porath

The Mighty

Maria Picone

TREND Community

Ying Qian

SMA Foundation

Sanath Kumar Ramesh

Open Treatments Foundation

Amy Raymond, PhD, PMP

Worldwide Clinical Trials

Deborah Requesens, PhD

UPenn Orphan Disease Center

Charlene Son Rigby

Global Genes

Steve Rodems, PhD

Travere Therapeutics

Kari Rosbeck

TSC Alliance

Luke Rosen, MS


Jean-Pascal Roussy


Joni Rutter, PhD

National Center for Advancing Translational Sciences (NCATS)

Christina SanInocencio, PhD, CPH, CNP

Fairfield University

Jillian Shaw

Broad Institute

Eric Sid, MD, MHA

National Center for Advancing Translational Sciences (NCATS)

Jennifer Sills

CSNK2A1 Foundation

Adam Staffaroni, PhD

Emily Stauffer

EveryLife Foundation

Kim Stephens, DBA

Dr. Joseph Muenzer MPS Research and Treatment Center at UNC, Chapel Hill

Ronda Thorington

Parent Coach

Stephanie Tomlinson

Undiagnosed Diseases Network

Karmen Trzupek, MS, CGC

Global Genes

Tiina Urv

Rare Diseases Clinical Research Network (RDCRN)

Jay Vivian, PhD

Children’s Mercy Kansas City

Vanessa Vogel-Farley

Global Genes

Shannon Von Felden

EveryLife Foundation

Yael Weiss

Mahzi Therapeutics

Ashley Winslow, PhD

Odylia Therapeutics

Kasey Woleben

Rare Village Foundation

Stephanie Yin


Advocate Support Program

The Advocate Support Program is offered to offset the cost associated with attending the 2023 RARE Advocacy Summit. Advocate Support will cover a portion of your meeting and travel expenses for the conference.

Advocate Support (formerly known as stipends) is available to individual patients, close family and friends of advocates in the rare disease community, or staff (paid/volunteer) of rare disease nonprofit organizations or support groups.  

Poster Submissions

Posters provide an opportunity for you to share the work of your organization, team or community with a larger audience of rare disease stakeholders from advocacy, research and industry. Showcasing ongoing or completed projects often can provide new connections/opportunities to meet new collaborators and/or potential funders. If you are interested in submitting a poster, click the button below to see poster guidelines for the RARE Advocacy Summit.

Visit the Exhibitor Booths

Exhibitors at RARE Advocacy Summit are featured here!

Applications for Exhibitor Booths have closed. While you are at the Summit, feel free to visit the booths! Suggested times to visit:

Tuesday, September 19, 1:00 pm -6:00 pm

Wednesday, September 20, 8:30 am – 6:00 pm
Make sure you stop by during the lunch break from 12:45 – 2:00 pm!

Thursday, September 21, 7:30 am-9:00 am

Plan Your Visit

Book your hotel room before August 23rd to secure a special conference rate. All conference room block reservations are in the Marina Tower.

Airfare Discounts: Delta and United are partnering with Global Genes to offer attendees discounted airfare (domestic travel only)!

Delta: Call Delta Meeting Network® at 1.800.328.1111* Monday–Friday, 8:00 a.m. – 6:30 p.m. (EST) and refer to Meeting Event Code NM2KV or click the button below.

United: Call United Meetings Reservation Desk Monday – Friday at (800) 426-1122 and use discount code ZNJJ289948 or click the button below.

Complimentary airport shuttle service to and from San Diego International airport runs daily every 30-40 minutes from 4:45 AM – 12:00 AM. Confirm next available time with the front desk. If you’re driving, self-parking is available ($27 per night). Accessible transportation is available upon request by contacting the hotel directly.

A Look Back at the 2022 Patient Advocacy Summit

2022 Recap

Check out some stats from our event last year.

2022 Rare Patient Advocacy Summit Stats

Thank You to Our Sponsors!

Champion Sponsor

Title Sponsors

Presenting Sponsor


Gold Sponsors

Chiesi global rare diseases

Silver Sponsors

Alliance Rx
Amicus Therapeutics
Avidity Biosciences
Jazz Pharmaceutical
Mallinckrodt Pharmaceuticals
Spark Therapeutics

Track Sponsors

Catalyst pharmaceuticals
Sangamo Therapeutics

Partner Sponsor


Advocacy Support Sponsors

Blueprint Medicines
Chiesi global rare diseases

Champions of Hope Support

Media Sponsor