The planning of this trial is the result of a strong collaborative effort between physicians, researchers, and patient advocates.
Dr. Michael Bober
Dr. Bober is a pediatrician and medical geneticist at the Alfred I. Dupont Hospital for Children in Wilmington, Delaware. He has a long involvement with the RCDP patient community and is in charge of a Registry for RCDP. Dr. Bober will act as the physician on both the future Natural History Study and PPI-1040 clinical trial.
dr. nancy braverman
Dr. Braverman is a medical geneticist and researcher at McGill University in Montreal, Quebec. She has been heavily involved with the Rhizokids community for over 10 years. Her lab focuses on understanding and evaluating treatments for peroxisomal disorders including RCDP. The Braverman lab is responsible for developing the RCDP1 mouse model used to test the ability to PPI-1040 to augment plasmalogen levels.
Shawn ritchie, PhD
Shawn is the CEO and Chief Scientific Officer at Med-Life Discoveries. He has over 17 years of experience using non-targeted metabolomics to investigate underlying biochemical causes of disease. Shawn is responsible for the strategic direction of MLD's diagnostics and therapeutics programs, including RCDP.
tara smith, PhD
Tara is the Vice President, Therapeutics at Med-Life Discoveries. She has worked for over 9 years on the pre-clinical assessment of plasmalogen precursors as potential therapeutic agents both in RCDP and diseases of aging. She oversees the day-to-day management of the therapeutics programs and maintains a close relationships with the all the involved consultants as well as Rhizokids International, the parent advocacy group.
cassie brown, MS, CCRC
Cassie is the research coordinator for the skeletal dysplasia program at Nemours- Alfred I. DuPont Hospital for Children in Wilmington, DE. She has spent three years organizing and implementing numerous research studies on skeletal dysplasias, including the Natural History Registry for RCDP. Cassie will act as the primary study coordinator for both the future natural history prospective trial and experimental therapy efficacy trial.
Melinda is the president of RhizoKids International, the largest parent advocacy group for patients with RCDP. She is the mother of 3 boys including Ethan, age 11, with RCDP. Melinda has been working with the team to provide information on what it is like to manage RCDP and what could be potential end-points worth evaluating in the trial.
Tracey is the co-founder of Rhizokids International, the largest parent advocacy group for patient with RCDP. She formed Rhizokids as an organization to raise funds and awareness to support those with RCDP and research towards a treatment. She is the mother of 2 boys including Jackson who was born with RCDP in 2007 and unfortunately passed away in 2013. She works tirelessly to advocate and support those walking the RCDP path and provides invaluable insight into RCDP and the RCDP community.
dan levy, PHD
Dan is the founder of DEL Biopharma, and brings over two decades of synthetic GMP manufacturing experience to the team. Dan is ultimately responsible for the synthesis and formulation efforts of PPI-1040.
dushmanthi jayasinghe, MSC
Dushmanthi is a Senior Analytical Chemist at MLD. She has been intimately involved in the initial synthesis of plasmalogen precursors, and is responsible for all method development analytical work at MLD including the finger-prick plasmalogen method being developed specifically for the RCDP trial.
Mousumi Bose, PhD
Mousumi is an Assistant Professor in the Department of Nutrition and Food Studies at Montclair State University. Having lost a child to Zellweger spectrum disorder (a rare disease closely related to RCDP), Mousumi’s research interests are based in understanding quality of life and other family-reported outcomes in rare pediatric diseases. She will be leading the development of a caregiver-reported survey tool to measure quality of life in children affected by RCDP.