Speaker Bios

Day of Hope - Science

Cystinosis Research Foundation – The Path to Better Treatments and a Cure

University of California, San Diego | La Jolla, CA
Dr. 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

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

Bambino Gesù Children’s Hospital | Rome, Italy 
Dr. 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 

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.

Massachusetts General Hospital | Boston, MA
Dr. 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. 

Stem Cell Clinical Trial Patient #1 | Consort, Alberta, Canada

Stem Cell Clinical Trial Patient #3 | Bremerton, WA

Day of Hope - Children's Program

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

University of Utah Hospital | Salt Lake City, UT
Dr. Jenkins graduated from the University of Utah School of Medicine in 2014. He works in Salt Lake City, UT and two other locations and specializes in Internal Medicine. Dr. Jenkins is affiliated with the University of Utah Hospital.
Dr. Jenkins is a member of the Cystinosis Research Foundation Scientific Review Board

Alberta Children’s Hospital, Calgary | Calgary, Alberta, Canada
Dr. Midgley’s medical training began in the UK at Cambridge University and then Oxford Medical School. He continued his pediatric training in various hospitals in the UK including a year in pediatric kidney transplantation at Guy’s Hospital in London, UK. Following a nephrology fellowship at the Hospital for Sick Children in Toronto in 1994, Dr. Midgley moved west to Calgary where he was recruited as the first pediatric nephrologist at the Alberta Children’s Hospital. In Calgary, Dr. Midgley has developed, along with five colleagues, a complete pediatric service for Southern Alberta including a busy chronic kidney disease, dialysis and transplant program.
Dr. Midgley is a member of the Cystinosis Research Foundation Medical and Scientific Advisory Board

St. Christopher’s Hospital for Children | Philadelphia, PA 
Dr. Zaritsky is a Pediatric Nephrologist who has been in practice for over 15 years. He received his MD and PhD from Stanford University School of Medicine and completed his fellowship in Pediatric Nephrology at UCLA. He has a history of basic and clinical research in iron metabolism. Dr. Zaritsky specializes in a wide range of kidney disorders, from common disorders such as kidney stones and nephrotic syndrome to rare disorders such as cystinosis and X-linked hypophosphatemia.  He has recently focused on the treatment of FSGS and is recognized by NephCure Kidney International as a Nephrotic Syndrome Specialist. Dr. Zaritsky is the Nephrology Section Chief at St. Christopher’s Hospital for Children and Professor of Pediatrics at Drexel College of Medicine in Philadelphia, Pennsylvania. 

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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