We are excited to announce that the Day of Hope conference videos are now available in French & Italian! To see the translations, select the video you’d like to watch, click the settings icon on the bottom right of your video screen and select the French or Italian subtitles.
Therapies in Cystinosis
What's New and What's Hot!
CRF Day of Hope
Science Conference
Thursday, April 29, 2021
9:00 am - 12:00 pm PDT
Zoom Webinar
Thank you for joining our research-focused Day of Hope Science webinar! We have posted a video of each presentation for you to enjoy. The presentations will be translated into French and Italian in the next few weeks.
The research presentations by Stéphanie Cherqui, PhD, Francesco Emma, MD, Morgan Fedorchak, PhD, Paul Grimm, MD, and Reza Seyedsadjadi, MD, provided the hundreds of attendees with updates on the progress and status of new therapies in cystinosis, what’s new in kidney transplantation, current development of the controlled release eye drop, progress in the study of myopathy and dysphagia, and current outcomes of the three patients in stem cell and gene therapy trial. We are grateful to our CRF-funded researchers, scientists and clinicians whose tireless work has helped transform the understanding and treatment of cystinosis.
We look forward to seeing you in person at the next CRF Day of Hope Conference at the Balboa Bay Club, Newport Beach, CA on Thursday, March 31 – April 2, 2022!
Cystinosis Research Foundation – The Path to Better Treatments
and a Cure
Nancy J. Stack, President and Founder
Strategies to Develop New Therapies in Cystinosis
Francesco Emma, MD | Bambino Gesù Children’s Hospital | Rome, ITALY
Moderated by Jill Emerson
Update on Transplantation, What’s New and What’s Hot!
Paul C. Grimm, MD | Stanford University School of Medicine | Stanford, CA
Moderated by Denice Flerchinger
The Development of Controlled Release Cysteamine Eye Drops
Morgan Fedorchak, PhD | University of Pittsburgh School of Medicine | Pittsburgh, PA
Moderated by Marcu Alexander
Myopathy and Dysphagia in Adults with Cystinosis
Reza Seyedsadjadi, MD | Massachusetts General Hospital | Boston, MA
Moderated by Stephen Jenkins, MD
Stem Cell and Gene Therapy Treatment – Update on the Trial and Patients
Stéphanie Cherqui, PhD | University of California, San Diego | La Jolla, CA
Moderated by Teresa and Kevin Partington
Life Post Stem Cell Transplant – Q&A with Jordan Janz and Jacob Seachord
Jordan Janz | Stem Cell Clinical Trial Patient #1 | Consort, Alberta, CANADA
Jacob Seachord | Stem Cell Clinical Trial Patient #3 | Bremerton, WA
Moderated by Teresa and Kevin Partington
Conclusion and closing remarks
Nancy J. Stack, President and Founder
CRF Board Member Moderators
Marcu Alexander | Hearts for Hadley I Boise, ID
Jill Emerson, CPA | Hope for Brooke | Hammonton, NJ
Denice Flerchinger | Tina’s Hope For a Cure | Clarkston, WA
Stephen Jenkins, MD | University of Utah Hospital | Salt Lake City, UT
Teresa and Kevin Partington | Jenna and Patricks’ Foundation of Hope | Sacramento, CA
Brian Sturgis | 24 Hours for Hank | Sandpoint, ID
CRF Day of Hope
Children’s Program
Saturday, May 1, 2021
9:00 am - 11:00 am PDT
Zoom Meeting
A big THANK YOU to our global cystinosis community; we were thrilled to see so many families from around the world participate in our first-ever virtual Day of Hope conferences! The conferences were a huge success and attended by families from 21 countries who joined the conference to learn more about the progress and status of our CRF-funded researchers.
The Saturday program featured an easy-to-understand presentation about cystinosis by Stephen Jenkins, MD, breakout sessions for parents of children with cystinosis including a private teen breakout session, and then a surprise interactive session with all of the children and their pets! We are pleased to include cystinosis experts and clinicians Paul Grimm, MD, Julian Midgley, MD, and Joshua “JJ” Zaritsky, MD, PhD.
We look forward to seeing you at the next CRF Day of Hope Conference at the Balboa Bay Club, Newport Beach, CA on Thursday, March 31 – April 2, 2022!
CRF Introduction and Welcome
During this session, participants will have the opportunity to briefly introduce their families.
Teresa and Kevin Partington
How to Teach Your Toddler and Your Grandparents About Cystinosis
Dr. Jenkins will lead a “Lego” presentation that demonstrates how parents and children with cystinosis can teach others about cystinosis.
Stephen Jenkins, MD
Family Breakout Sessions
During these sessions, participants will have the opportunity to ask the cystinosis experts questions via the Chat function. Children’s sessions are geared toward parents (children may accompany their parents in the breakout session if desired). Teens’ session is for teens only, and Parents of Teens for parents only.
Parents of Little Ones (0–5 yrs)
Hosted by Joshua “JJ” Zaritsky, MD, PhD
Moderated by Jill Emerson
Parents of Kids (6–12 yrs)
Hosted by Julian Midgley, BM, BCh (MD)
Moderated by Stephen Jenkins, MD
Teens (13 and up)
Hosted by Paul C. Grimm, MD
Moderated by Lauren Hartz
Parents of Teens
Hosted and Moderated by Brian Sturgis
Show and Tell Us About Your Pets!
Meet some of the pets in our CRF community during this fun social session! If you don’t have any pets to share, you may share something special with the group.
Marcu Alexander
Conclusion
Wrap-up session with closing remarks.
Denice Flerchinger
CRF Board Member Program Co-Chairs
Jill Emerson, CPA | Hope for Brooke | Hammonton, NJ
Stephen Jenkins, MD | University of Utah Hospital | Salt Lake City, UT
Brian Sturgis | 24 Hours for Hank | Sandpoint, ID
