Thursday, March 31, through Saturday, April 2, 2022

Balboa Bay Resort, 1221 W. Coast Highway, Newport Beach, CA

On March 31, 2022, cystinosis families gathered once again to learn, laugh, share, and reconnect at the Day of Hope Conference in Newport Beach. The conference was a weekend of hope and inspiration, where we renewed our friendships, created new friendships, and recommitted our efforts to work together to support research that will improve the lives of our children and adults with cystinosis.

CRF-funded researchers and clinicians presented their research updates and discussed the challenges of cystinosis. Families were brought up to speed on the research we fund and had the opportunity to ask questions from the experts. The conference sessions were recorded and are now available for viewing.

The recorded sessions include an update by Morgan DiLeo, PhD, on the novel eye treatment for ocular cystinosis, an exciting update from Beno Freedman, PhD, on his research news using iPS cells to develop therapeutics for kidney disease, and a fascinating presentation by Veenita Khare, PhD, about advancing our understanding of renal Fanconi Syndrome.

Information about the Day of Hope Conference is listed below along with the video presentations, and topic titles. 

Ocular Biodistribution of Cysteamine Delivered by a Sustained Release Microsphere/ Thermoresponsive Gel Eye Drop
Watch Video
CCIR and You – Help Us Find A Cure
Watch Video
Using Human Pluripotent Stem Cells to Develop New Treatments For Cystinosis
Watch Video
Kidney Transplant Smorgasbord
Watch Video
Managing All Those Supplements When Your Child or You Have Fanconi Syndrome
Watch Video
Cystinosis: Back to the Basics
Watch Video
Advancing the Understanding of the Renal Fanconi Syndrome in Cystinosis
Watch Video
Muscle Wasting in Children with Cystinosis
Watch Video
The Varied Challenges of Cystinosis for Young Adults and Adults
Watch Video
Myopathy and Dysphagia in Adults with Cystinosis
Watch Video

Day of Hope Conference Overview

Thursday, March 31, 2022

3:00 pm – 8:00 pm Check-In
6:00 pm – 9:00 pm Dinner Reception (Indoor/Outdoor Dining)  

Friday, April 1, 2022

6:30 am – 8:00 am Breakfast – Terrace Outdoor Dining
7:30 am – 12:00 pm Childcare (5-12 years) Bayfront Lawn
8:00 am – 12:00 pm Morning Conference Sessions
12:00 pm – 1:00  pm
Lunch – Terrace Outdoor Dining
1:00 pm – 4:30 pm Childcare Resumes Quarterdeck Room
1:00 pm – 4:30 pm Afternoon Conference Sessions
6:00 pm – 9:00 pm Family Dinner Outdoor Dining  

Saturday, April 1, 2022

6:30 am – 8:00 am Breakfast – Ballroom Terrace Outdoor Dining 
7:30 am – 12:15 pm Childcare Quarterdeck Room 
8:00 am – 12:00 pm Morning Conference Sessions
12:15 pm – 2:00 pm
Lunch  
Conference Presenters with Abstracts PDF

Clinics and Information

We encourage you to review information about each of the clinics represented at the conference and contact the representatives for further information. 

Cure Cystinosis International Registry (CCIR):  Volunteers Needed
Join the 130 families from around the world who and enroll in the patient registry. When you enroll you are helping the research community learn more about cystinosis so they can target research that will lead to better treatments and a cure. Do your part! Visit with Clay Emerson to learn more and enroll.

Confocal Clinic:  Volunteers Needed for Skin Imaging
Dr. Cherqui is conducting a research study to monitor the natural history of cystinosis and do skin confocal imaging. If you have questions and/or are interested in participating, please contact Imama Ahmed (i1ahmed@health.ucsd.edu) or Anne Sawyers (amsawyers@health.ucsd.edu).

Muscle Wasting:  Volunteers Needed for blood draws, height & weight measurements
Dr. Mak and Dr. Cherqui are conducting a research study to monitor the natural history of cystinosis and to draw blood. If you have any questions and/or are interested in participating, please contact Kristin Zeeb (kzeeb@health.ucsd.edu)  or Anne Sawyers (amsawyers@health.ucsd.edu).

Distal Myopathy and Dysphagia:  Volunteers Needed to sign up (actual trial is in Boston)
Please stop by the table and talk with Natalie Grant and Stacey Sullivan. Information about the study can be downloaded at https://www.cystinosisresearch.org/current-clinical-trials/.

 

CRF Day of Hope Family Conference

Thursday, March 31, through Saturday, April 2, 2022

Balboa Bay Resort, 1221 W. Coast Highway, Newport Beach, CA

Learn, share, laugh, and celebrate our cystinosis community at the Day of Hope family conference. We will renew our friendships and our efforts to work together to support research that will improve the lives of our children and adults with cystinosis.

The Day of Hope will begin with a welcome Mexican Fiesta Dinner on Thursday evening, March 31, and conference sessions will begin Friday morning, April 1, and continue through noon on Saturday, April 2.

CRF-funded researchers including Sergio Catz, PhD, Stéphanie Cherqui, PhD, Morgan DiLeo, PhD, and others will present their current research findings and report on the progress of their studies. Paul Grimm, MD, and Stephen Jenkins, MD, will join us to discuss cystinosis treatment including renal replacement therapy. There will be breakout sessions that will allow us to discuss life with cystinosis and how we can support and learn from each other. We have so much to share; we hope you will join us to celebrate our research progress and our community! Watch for more details this week!

Day of Hope Co-Chairs

Jill Emerson
Stephen Jenkins, MD
Teresa Partington
Nancy Stack
Brian Sturgis

COVID-19 Policy Update The CDC (Centers for Disease Control)

The CDC has lifted the mask mandate in areas where transmission of COVID-19 is Low.  Orange County, California is in the Low category. “People in the Low areas may choose to wear a mask at any time. People with symptoms, a positive test, or exposure to someone with COVID-19 should wear a mask.” 

The State of California has issued the following statement: 
“Starting March 1, masks will no longer be required for unvaccinated individuals but will be strongly recommended for all individuals in most indoor settings. After March 11, in schools and child care facilities, masks will not be required but will be strongly recommended. Masks will still be required for everyone in high transmission settings like public transit, emergency shelters, health care settings, correctional facilities, homeless shelters and long-term care facilities.”

CRF will follow the guidance from the CDC and the State of California and allow people to choose to wear a mask. That said, because we have a vulnerable population of children and adults, we strongly recommend that masks be worn in indoor settings. We are still requiring that all conference attendees age 5 years and older be fully vaccinated. We will have an ample supply of masks and hand sanitizer available throughout the conference venue. We look forward to seeing you soon!