Renewing Hope, Rekindling Connections, Realizing a Brighter Tomorrow
Thursday, April 10 through Saturday, April 12, 2025
Hyatt Regency Beach Resort and Spa in Huntington Beach, CA
CRF Day of Hope Family Conference
Day of Hope Information
The Day of Hope conference will begin on Thursday, April 10, with registration and a family Welcome Session followed by a Mexican Fiesta Dinner. Conference sessions will begin Friday morning, April 11, and continue through Saturday, April 12, and conclude with a sunset BBQ dinner on Saturday evening.
CRF-funded researchers Sergio Catz, PhD, and Stéphanie Cherqui, PhD, 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 treatments, including renal replacement therapy. We have planned multiple 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 cystinosis community!
Conference Overview
THURSDAY AFTERNOON
Registration Opens at 3:00 p.m.
Welcome Session 4:00 p.m.
Mexican Fiesta Dinner 6:00 p.m.
FRIDAY
Family Breakfast
Children’s Activities
Conference Sessions
Family Lunch
Conference Sessions
Dinner Under the Stars
SATURDAY
Family Breakfast
Children’s Activities
Conference Sessions
Family Lunch
Conference Sessions
Sunset BBQ Family Dinner
Conference Agenda
Thursday, April 10
3:00 PM – 8:00 PM
Conference Registration Check-in
Grand Foyer
4:00 PM – 6:30 PM
Childcare Hours
Salon E (ages 0 – 3)
Salon F (ages 4 – 7)
Salon G (ages 8 – 12)
4:30 PM – 6:30 PM
Family Introductions
Theater Salon D
Ben and Marcu Alexander, CRF Trustees
5:45 PM – 6:30 PM
Teens and Tweens Introductions (ages 12-17)
The Cove Lounge
6:30 PM – 9:00 PM
Day of Hope Welcome Dinner Reception
California Courtyard & The Cove Patio
Friday, April 11
7:00 AM – 8:30 AM
Breakfast
Grand Foyer South & Terrace
8:15 AM – 12:00 PM
Childcare Hours
Salon E (ages 0 – 3)
Salon F (ages 4 – 7)
Salon G (ages 8 – 12)
Presentation Sessions Theater Salon D
Moderator: Clay Emerson, PhD, CRF Trustee
8:30 AM – 8:50 AM
Cystinosis Research Foundation: A Community United for A Cure
Nancy Stack, President, Cystinosis Research Foundation
8:55 AM – 9:15 AM
What’s New in Cystinosis Research?
Stephen Jenkins, MD, CRF Trustee
University of Utah Hospital
9:00 AM – 1:00 PM
Teens and Tweens Fieldtrip to Dr. Albrecht’s Lab (ages 12 – 17)
Bus departs from the Grand Foyer
9:20 AM – 9:40 AM
Cystinosis, The Early Years: Fanconi Syndrome and More
Laurence Greenbaum, MD, PhD, FAAP
Emory University School of Medicine and Children’s Healthcare of Atlanta
9:45 AM – 10:05 AM
Planning Your Life in Relation to Your Kidney Function, i.e. Planning for Kidney Transplant, and Update on GI Issues
Paul C. Grimm, MD
Stanford University School of Medicine and Stanford Children’s Health
10:10 AM – 10:30 AM
Living Well with Cystinosis After Kidney Transplantation
Julian Midgley, BM BCh
Alberta Children’s Hospital, University of Calgary
10:35 AM – 12:00 PM
Adults with Cystinosis and Partners
Sunrise and Sunrise Foyer
Discussion Leaders: Shannon Henderson and Natalie Stack Morgan
10:35 AM – 10:50 AM
Break
Grand Foyer
Break Out Sessions Various Locations
10:55 AM – 12:00 PM
Parents of Children (ages 0 – 4)
Bolsa Chica Boardroom
Discussion Leader: Jill Emerson, CRF Trustee and Ryan Hub, Paul Grimm, MD
10:55 AM – 12:00 PM
Parents of Children (ages 5 – 8)
Salon B
Discussion Leaders: Kristen Murray and Brian Sturgis, CRF Trustees,
Julian Midgley, BM BCh
10:55 AM – 12:00 PM
Parents of Tweens (ages 9 – 12)
Salon C
Discussion Leaders: Clay Emerson, CRF Trustee, Laurence Greenbaum, MD, PhD
10:55 AM – 12:00 PM
Parents of Teens (ages 13 – 17)
Royal Tern
Discussion Leaders: Marcu Alexander and Stephen Jenkins, MD, CRF Trustees, Francesco Emma, MD
10:55 AM – 12:00 PM
Parents of Adults with Cystinosis
Salon A
Discussion Leaders: Denice Flerchinger and Nancy Stack
10:55 AM – 12:00 PM
Grandparents
Driftwood
Discussion Leaders: Bill and Diane Neumann
12:00 PM – 1:00 PM
Lunch
Grand Foyer South & Terrace
12:50 PM – 4:45 PM
Childcare Hours
Salon E (ages 0 – 3)
Salon F (ages 4 – 7)
Salon G (ages 8 – 12)
1:00 PM – 6:00 PM
Teens and Tweens Private Day at the Cabana (ages 12 – 17)
Slyders Cabanas (Kids Pool Area – Cabanas 3 & 4)
Presentation Sessions Theater Salon D
Moderator: Stephen Jenkins, MD, CRF Trustee
1:05 PM – 1:15 PM
Cure Cystinosis International Registry (CCIR)
Clay Emerson, PhD, CRF Trustee
1:20 PM – 1:40 PM
Neuromarkers of Sensory and Cognitive Function in Cystinosis
Sophie Molholm, PhD
Albert Einstein College of Medicine
1:45 PM – 2:05 PM
Effects of a Ketogenic Diet on Murine Models of Cystinosis
Francesco Emma, MD
Bambino Gesù Children’s Hospital, Rome, Italy
2:10 PM – 2:40 PM
Medical Panel
Stéphanie Cherqui, PhD, Francesco Emma, MD, Paul Goodyer, MD,
Laurence Greenbaum, MD, PhD, Paul Grimm, MD, Stephen Jenkins, MD,
Julian Midgley, BM BCh, Reza Seyedsadjadi, MD
2:45 PM – 3:00 PM
Break
Grand Foyer
3:05 PM – 3:15 PM
Living Life to the Fullest: Our Journey with Cystinosis
Hadley and Stella Alexander
3:20 PM – 4:00 PM
Fostering Resilience in Ourselves and Others
Kristin Van Marter-Souers, MA, LMHC
Kristin Souers, LLC – fosteringresiliantlearners.org
4:05 PM – 4:20 PM
What Have We Learned Today?
Stephen Jenkins, MD, CRF Trustee
6:00 PM – 9:00 PM
Dinner Under the Stars
Lighthouse Courtyard
Saturday, April 12
7:00 AM – 8:30 AM
Breakfast
Grand Foyer South & Terrace
8:15 AM – 12:00 PM
Childcare Hours
Salon E (ages 0 – 3)
Salon F (ages 4 – 7)
Salon G (ages 8 – 12)
Presentation Sessions Theater Salon D
Moderators: Kevin and Teresa Partington, CRF Trustees
8:30 AM – 8:40 AM
Welcome
CRF Trustee: Brian Sturgis
8:45 AM – 9:05 AM
Applying the COVID mRNA Technology to Treatment of Cystinosis
Paul R. Goodyer, MD
McGill University
9:10 AM – 9:40 AM
Phase 1/2 Clinical Trial of Autologous Hematopoietic Stem and Progenitor Cell Gene Therapy for Cystinosis
Stéphanie Cherqui, PhD
University of California, San Diego
9:45 AM – 9:55 AM
The Story Behind the Wish: Living with a Rare Disease
Natalie Stack Morgan
10:00 AM – 10:30 AM
Design of Ph1/2 CYStem Study of Ex Vivo HSC Gene Therapy in Nephropathic Cystinosis
Daniel Grant, Vice President & Global Program Head
Novartis Global Drug Development, Neuroscience & Gene Therapies Unit
10:35 AM – 10:50 AM
Break
Grand Foyer South & Terrace
Presentation Sessions Theater Salon D
Moderator: Denice Flerchinger, CRF Trustee
10:55 AM – 11:15 AM
Targeting the Lysosomal-Methylation Signaling Axis in Cystinotic Bone and Kidney Disease
Lauren Albrecht, PhD
University of California, Irvine
11:20 AM – 11:55 AM
Breakout: Adults with Cystinosis and Partners
Sunrise and Sunrise Foyer
Discussion Leaders: Shannon Henderson, Natalie Stack Morgan,
Paul Grimm, MD, Reza Seyedsadjadi, MD, Donny Suh, MD, FAAP, MBA, FACS
11:20 AM – 11:55 AM
Breakout: Only Teens and Tweens with Cystinosis (ages 12 – 17)
Shoreline
Discussion Leader: Stephen Jenkins, MD, CRF Trustee
Presentation Sessions Theater Salon D
11:20 AM – 11:30 AM
Impact of Cystinosis on Bone Health
Isidro Salusky, MD
David Geffen School of Medicine at UCLA, Los Angeles
11:35 AM – 11:55 AM
Translational Approaches for Cystinosis Targeting Autophagy
Sergio D. Catz, PhD
The Scripps Research Institute
12:00 PM – 1:00 PM
Lunch
Lighthouse Courtyard
12:50 PM – 3:30 PM
Childcare Hours
Salon E (ages 0 – 3)
Salon F (ages 4 – 7)
Salon G (ages 8 – 12)
1:00 PM – 3:00 PM
Teens and Tweens Boardwalk Bike Ride and Bites (ages 12 – 17)
Teens Meet at Registration Table and Depart from Grand Foyer
Presentation Sessions Theater Salon D
Moderator: Kristen Murray, CRF Trustee
1:10 PM – 1:30 PM
Ocular Manifestations of Cystinosis You Can’t Afford to Miss!
Donny Suh, MD, FAAP, MBA, FACS
Gavin Herbert Eye Institute (GHEI) & Fellowship Program & Eye Mobile Program
University of California, Irvine
1:35 PM – 1:55 PM
Clinical Trial for Swallowing Difficulty in Adult Patients with Cystinosis
Reza Seyedsadjadi, MD
Massachusetts General Hospital, Harvard Medical School
2:00 PM – 2:35 PM
Adult Panel
Geoffrey Couppey, Tina Flerchinger, Kurt Gillenberg, Shannon Henderson, Mack Maxwell, Jenna Partington, Patrick Partington, Kathleen Roberts, Jacob Seachord, Natalie Stack Morgan, Alex and Bryan Stout, Henry Sturgis, Brandon Waldron, Heather Wegerif
2:40 PM – 3:00 PM
Conference Wrap-Up
Day of Hope Co-Chairs: Jill Emerson, Clay Emerson, Stephen Jenkins, Kristen
Murray, Brian Sturgis, CRF Trustees
6:00 PM – 9:00 PM
Closing Dinner Celebration
Lighthouse Courtyard
Speaker Abstracts
Targeting the Lysosomal-Methylation Signaling Axis in Cystinotic Bone
and Kidney Disease
Lauren Albrecht, PhD
University of California, Irvine
Translational Approaches for Cystinosis Targeting Autophagy
Sergio D. Catz, PhD
The Scripps Research Institute
Phase 1/2 Clinical Trial of Autologous Hematopoietic Stem and Progenitor Cell Gene Therapy for Cystinosis
Stephanie Cherqui, PhD
University of California, San Diego
Effects of a Ketogenic Diet on Murine Models of Cystinosis
Francesco Emma, MD
Bambino Gesù Children’s Hospital, Rome, Italy
Repurposing COVID Vaccine mRNA Technology for Therapy of Cystinosis
Paul R. Goodyer, MD
McGill University
Cystinosis, The Early Years: Fanconi Syndrome and More
Laurence Greenbaum, MD, PhD, FAAP
Emory School of Medicine
Children’s Healthcare of Atlanta
Planning Your Life in Relation to Your Kidney Function, i.e., Planning for Kidney Transplant, and Update on GI Issues
Paul C. Grimm, MD
Stanford University School of Medicine and Stanford Children’s Health
What’s New in Cystinosis Research?
Stephen Jenkins, MD
University of Utah Health
CRF Board of Trustees and Scientific Review Board
Living Well with Cystinosis After Kidney Transplantation
Julian Midgley, BM BCh
Alberta Children’s Hospital, University of Calgary
Neuromarkers of Sensory and Cognitive Function in Cystinosis
Sophie Molholm, PhD
Albert Einstein College of Medicine
Impact of Cystinosis on Bone Health
Isidro B. Salusky, MD
David Geffen School of Medicine at UCLA, Los Angeles
Clinical Trial for Swallowing Difficulty in Adult Patients with Cystinosis
Reza Seyedsadjadi, MD
Massachusetts General Hospital
Harvard Medical School
Ocular Manifestations of Cystinosis You Can’t Afford to Miss!
Donny W. Suh, MD, FAAP, MBA, FACS
Gavin Herbert Eye Institute (GHEI) &
Fellowship Program & Eye Mobile Program
University of California at Irvine
Fostering Resiliance in Ourselves and Others
Kristin Van Marter-Souers, MA LMHC
Kristin Souers, LLC
Cystinosis Research Foundation
Day of Hope 2025 Co-Chairs
Clay Emerson, PhD, PE, CMF, Hope for Brooke, CRF Board Member
Jill Emerson, CPA, Hope for Brooke, CRF Board Member
Stephen Jenkins, MD, University of Utah Hospital, Sam's Hope for a Cure, CRF Board Member
Kristen Murray, Seth's Circle of Hope, CRF Board Member
Brian Sturgis, 24 Hours for Hank, CRF Board Member
We are excited to learn, share, laugh, and celebrate our cystinosis community at the Day of Hope family conference. We will renew our friendships and our united efforts to support research that will improve the lives of our children and adults with cystinosis.
We look forward to seeing you soon!
Questions email Jill Emerson, jill.emerson@hotmail.com
Hyatt Regency Huntington Beach Resort and Spa
General Accommodation Information
The 2025 Day of Hope Conference will be held at the Hyatt Regency Huntington Beach Oceanfront Resort and Spa, on the beautiful coastal shores of Huntington Beach. We look forward to welcoming our cystinosis families and encourage you to register as soon as you can to guarantee your attendance and ensure your hotel accommodations.
The Day of Hope Committee has secured a block of rooms at a special discounted rate of $349 per night, plus taxes and fees for all attendees. Cystinosis families, including grandparents, will receive a $125 credit* per night towards their total bill, courtesy of the Day of Hope sponsors. Conference registration is required to secure the discounted rate and will only be applied to bookings utilizing the dedicated Day of Hope lodging link (provided upon successful registration submission). Rates are available for a maximum of four (4) nights. Guests are responsible for all incidentals and parking expenses incurred. Check-in time is 4:00 p.m. and check-out is 11:00 a.m.
There is no charge to attend the conference, and all group meals provided Thursday evening through Saturday evening are included. Childcare and related activities are provided by licensed and bonded sitters for infants and children up to 12 years of age during conference sessions, and are complimentary for all families (pre-registration is required – details are available upon successful registration submission).
*NOTE: The discounted room rate for all attendees is $349.00 plus taxes and fees of $74.29 (for a total nightly rate of $423.29). Cystinosis families and grandparents will receive a $125.00 credit per night courtesy of Day of Hope sponsors (for a nightly rate of $298.28). The credit will be applied by the hotel at the time of departure and will NOT be reflected when booking your room. All room rates quoted exclude room upgrades, incidentals, and parking charges.
