Rare Patient Advocacy Symposium



Supported by


May 6, 2016

Sheraton University City Hotel
3549 Chestnut Street
Philadelphia, PA 19104

Date and Time

Date: Friday, May 6th, 2016


Program Presentation Materials



This first-time collaboration between Global Genes and the University of Pennsylvania Orphan Disease Center (ODC) capitalizes on the Million Dollar Bike Ride (MDBR) event taking place on May 7 in which teams supporting different rare diseases ride to raise money for research projects that benefit their communities.

The Symposium is open to all patients and caregivers affected by rare disease and who would like to come together for a half-day program on Friday, May 6, the day before the MDBR. Attendees don’t need to be affiliated with the MDBR and all rare disease patients and advocates from the surrounding area are welcome to attend. Attendance for patients/advocates is $20.00 and all registration dollars are evenly split between Global Genes and ODC to support the Global Genes RARE Patient Impact Grant Program and the research grants for rare diseases given by ODC as part of the MDBR.

This program is made possible with support from REGENXBIO.



• Patient Engagement in Drug Development

• Building a Plan to Pursue Research

• Finding and Raising Funds for Research

• Organizational Growth


Registration and Agenda

Registration for advocates for the program is $20.00, and all registration proceeds will be split evenly to support the Global Genes RARE Patient Impact Grant Program and Penn Medicine’s Million Dollar Bike Ride program that funds research. For questions about the program, please reach out to Kym Kilbourne at kymk@globalgenes.org.
Should the registration cost be a barrier to advocates, please contact Samantha Charleston at scharle@mail.med.upenn.edu.


You can view the RARE Patient Advocacy Symposium agenda here.


About Million Dollar Bike Ride

The Million Dollar Bike Ride, now in its third year, brings more 600 cyclists and volunteers to Penn’s campus to ride either 12, 33, or 73 miles starting in the city, and ranging across the Greater Philadelphia region. In two years, the MDBR’s 20+ teams have raised more than $2 million to fund research grants on the diseases they represent.  The ODC ensures that 100% of the funds raised goes towards these pilot grants, with no overhead taken out.

All funds raised by Disease Teams through pledges are used by the ODC to expand the scope and depth of the pilot grant program.  Following the event, a Request for Application (RFA) will be broadly distributed to the international scientific community requesting pilot grant applications to study diseases for which designated money has been raised. The ODC is securing philanthropic and corporate donations that will be used to match dollar‐for‐dollar money raised by the cycling participants. www.milliondollarbikeride.org


2016 MDBR Teams/Rare Diseases Represented
APBD Tour de Friends
A-T Children’s Project
Cure CMD
CDCN: Castleman Disease
Team CDLK5 Riding for a Cure: Int’l Foundation for CDKL5
CHI: Congenitl Hyperinsulinism
Team Cure ML4: Mucolipidosis type IV
Emily’s Entourage
Curing Retinal Blindness Foundation
Fibrous Dysplasia Foundation
Team Josh and DCO Riders: Dyskeratosis Congenita
The LAM Foundation
Lymphangiomatosis & Gorham’s Disease Alliance (LGDA)
Miles for Millie
Movin’ for Mallory – Cure Cystic Fibrosis
Team MPS: Mucopolysaccharidoses
NBIA Disorders
Team NPC (Niemann Pick Type C)
Team NTSAD: Tay-Sachs, Sandhoff, GM1 and Canavan
Parent Project Muscular Dystrophy
Penn Scientist for Orphan Disease Research
Pedal for Pitt Hopkins
RASopathies Network
The Ryan Foundation
Snyder-Robinson Syndrome
The Stop ALD Foundation