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! In the next few weeks, we will post a video of each presentation, with translations in French and Italian.  

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! 

9:00AM – 9:15AM  Cystinosis Research Foundation – The Path to Better Treatments and a Cure
Nancy J. Stack, President and Founder

9:15AM – 9:35AM  Strategies to Develop New Therapies in Cystinosis
Francesco Emma, MD | Bambino Gesù Children’s Hospital | Rome, ITALY
Moderated by Jill Emerson

9:40AM – 10:05AM  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

10:10AM – 10:30AM  The Development of Controlled Release Cysteamine Eye Drops
Morgan Fedorchak, PhD | University of Pittsburgh School of Medicine | Pittsburgh, PA
Moderated by Marcu Alexander

10:35AM – 10:55AM  Myopathy and Dysphagia in Adults with Cystinosis
Reza Seyedsadjadi, MD | Massachusetts General Hospital | Boston, MA
Moderated by Stephen Jenkins, MD

11:00AM – 11:25AM  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

11:25AM – 11:45AM  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

11:45AM – 12:00PM  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

We are excited to host our Children’s Day of Hope program to bring our families together for a fun meet-up and lots of conversation! The Saturday program will include a welcome and introduction, 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! We are pleased to include cystinosis experts and clinicians Paul Grimm, MD, Julian Midgley, MD, and Joshua “JJ” Zaritsky, MD, PhD.  Be ready to learn, laugh, share, and connect!  

9:00AM – 9:30AM  CRF Introduction and Welcome
During this session, participants will have the opportunity to briefly introduce their families.
Teresa and Kevin Partington

9:30AM – 9:45AM  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

9:45AM – 10:15AM  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

10:15AM – 10:45AM  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

10:45AM – 11:00AM   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 

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