CRF Day of Hope Science Conference Zoom Webinar Thursday, April 29, 2021 - 9:00 am - 12:00 pm PDT
Mark your calendars and plan on joining us for an update on a selection of CRF research projects. The conference will feature five CRF-funded researchers including Stéphanie Cherqui, PhD, Francesco Emma, MD, Morgan Fedorchak, PhD, Paul Grimm, MD, and Reza Seyedsadjadi, MD. Each live presentation will be followed by an interactive Q & A session. We will also have a special Q & A session with Jordan Janz, and Jacob Seachord, both patients in Dr. Cherqui’s stem cell transplant study. Jordan and Jacob will share their experiences and answer your questions about the trial.
We will have more information soon including registration details. We can’t wait to connect with you again and share the progress we have made in our quest for better treatments and a cure.
Day of Hope Virtual Science Conference Confirmed Speakers
- Cystinosis Research Foundation – The Path to Better Treatments and a Cure
Nancy J. Stack, President and Founder
- Stem Cell and Gene Therapy Treatment – Update on the Trial and the Patients
Stéphanie Cherqui, PhD | University of California, San Diego | La Jolla, CADr. Cherqui received her PhD in 2002 at Necker Hospital (Paris, France). Dr. Cherqui’s early research focused on the molecular characterization of cystinosis as well as the generation of mouse model. While at The Scripps Research Institute during her post-doctoral internship, she specialized in stem cells and gene therapy and was appointed Assistant Professor in 2009. In 2012, Dr. Cherqui joined the University of California, San Diego, Department of Pediatrics, Division of Genetics and became an Associate Professor in 2016. She established that hematopoietic stem cell (HSC) transplantation could reverse multi-organ degenerative disorders, even in the context of an intracellular membrane protein such as cystinosis. Her work led to the first in human HSC gene therapy clinical trial for cystinosis.
Dr. Cherqui is a member of the Cystinosis Research Foundation Scientific Review Board
- 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
- Update on Transplantation, What’s New, and What’s Hot!
Paul C. Grimm, MD | Stanford University School of Medicine | Stanford, CA
Dr. Grimm has been focused on care of children with renal disease. This has included basic and clinical research in transplant, dialysis, ambulatory hypertension and anemia related to renal disease. His early basic work has been focused on the pathogenesis of subclinical rejection, and chronic allograft damage (chronic rejection). Dr. Grimm cares for children who need or have received a renal transplant. He is the Medical Director of the Pediatric Kidney Transplant Program, the busiest pediatric kidney transplant program in the USA.
Dr. Grimm is a member of the Cystinosis Research Foundation Scientific Review Board
- Strategies to Develop New Therapies in Cystinosis
Francesco Emma, MD | Bambino Gesù Children’s Hospital | Rome, ITALYDr. Emma received his medical degree from the Catholic University of Louvain, Brussels, Belgium, where he specialized in Pediatrics. He subsequently completed his training in Pediatric Nephrology at Boston Children’s Hospital, Harvard Medical School. He moved to the Bambino Gesù Children’s Hospital in Rome in 1998 and was appointed Head of the Pediatric Nephrology Division in 2005. He currently holds the position of Head of the Department of Pediatric Subspecialties. Dr. Emma’s primary research interests lie in rare renal diseases, specifically in cystinosis and nephrotic syndrome.
Dr. Emma is a member of the Cystinosis Research Foundation Scientific Review Board
- Preclinical Testing and Next Steps for Controlled Release Cysteamine Eye Drop Morgan Fedorchak, PhD | University of Pittsburgh School of Medicine | Pittsburgh, PA
Dr. Fedorchak is the Director of the Ophthalmic Biomaterials Lab at the University of Pittsburgh, which focuses on the design and testing of various drug delivery systems and regenerative biomaterials, with an emphasis on ophthalmic applications. Dr. Fedorchak received her training in biomaterials fabrication, including surface conjugation and imaging, and rational design of controlled release systems. Dr. Fedorchak’s primary goal is to use her interdisciplinary experience and network of clinicians, basic scientists, and engineers to perform translational research in novel therapeutics. Her focus has been on bridging the gap between the clinical needs of patients and the engineering capabilities inherent to various biomaterials to expedite translation.
- Myopathy and Dysphagia in Adults with Cystinosis
Reza Seyedsadjadi, MD | Massachusetts General Hospital | Boston, MADr. Seyedsadjadi is a neurologist with a special interest in neuromuscular medicine and with research interests in disease outcomes and measures. Through collaboration with the Center for Rare Neurological Disorders (CRND) and Harvard Stem Cell Institute, he has also had the opportunity to work in further clinical characterization and clinical trial readiness of rare diseases primarily with neurological manifestations such as nephropathic cystinosis. Dr. Seyedsadjadi is the recipient of the Cystinosis Research Foundation awards for optimizing dysphagia assessments and further characterization of myopathy and dysphagia and evaluation for inherent muscle resilience and regenerative capacity.
Day of Hope Children's Program Saturday, May 1, 2021 9:00 am - 11:00 am PDT Virtual 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. Elements of past Day of Hope conferences will be included, but we will also create new experiences and interactions. Be ready to learn, laugh, share, and connect! More information and registration information coming soon!