CLINICAL TRIAL

CLINICAL TRIAL

We have partnered with the University of Florida College of Medicine and RESTEM LLC to to initiate an important trial for Duchenne muscular dystrophy (DMD).

Clinical Trial Overview

Philanthropic support has been critical to the center’s advancement of clinical trials. By partnering with the JAR of Hope Foundation and RESTEM LLC, the University of Florida's Center for Regenerative Medicine (CRM) will be able to initiate an important trial for Duchenne muscular dystrophy (DMD). UF has already been selected as the clinical trial site and RESTEM, LLC has received authorization from its Investigational New Drug Application to the FDA entitled “A phase 1, randomized, double-blind, placebo-controlled Cross Over study to assess the safety, tolerability, and preliminary efficacy of Umbilical Cord Lining stem cells (ULSC) in Pediatric patients with Duchenne’s muscular dystrophy (DMD)” to proceed with a Phase 1 trial in 12 patients. Additional support from the JAR of Hope Foundation will help translate Phase 1 and later Phase 2 trial with more answers as to if this will help these children with this debilitating disease. Applying the CRM’s research broadly, center leadership is seeing positive early results indicating that regenerative medicine might be a better course of treatment for some patients than immunosuppressive medicines with powerful side effects. 

RESTEM is an innovation-driven, clinical-stage, biotechnology firm, based in Corona, California, dedicated to the discovery, development and commercialization of novel, next-generation, cell-based therapeutics and tools that aid in the treatment of a broad range of degenerative disorders.


The company’s mission is focused on improving the quality of life for those who suffer from disabling diseases of the immune system, age-associated disorders, and other degenerative diseases. With over 14 years of research and development focused on the company’s novel and proprietary, umbilical cord lining stem cell, or ULSCs, which have novel regenerative properties believed to be superior to mesenchymal stem cells, RESTEM has emerged as a major contributor to cell-based treatments with ongoing clinical trials that address significant unmet medical need across a range of diseases and disorders.

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