~ Natural history studies of organic acidemias are needed for development of patient treatments ~
[Charlottesville, VA - April 21, 2016]-- The Organic Acidemia Association, together with HemoShear Therapeutics and Children's National Medical Center, today announced a grant from the National Organization for Rare Disorders (NORD) to begin natural history studies for organic acidemias, which are genetic metabolic diseases with life-threatening consequences. The grant from NORD is supported in part by a cooperative agreement with the U.S. Food and Drug Administration (FDA). Organic acidemias affect only a small number of children. There is often little information available regarding the course of the diseases and there are few, if any, therapeutic options.
"We are thrilled with the grant provided by NORD and the help they will provide us to better understand organic acidemias," said Kathy Stagni, executive Director of the Organic Acidemia Association and parent of a child with propionic acidemia. "We are hopeful that this information will pave the way for discovery of treatments for our children."
Natural history studies provide important information about the progression of a disease, which can be used to design clinical trials for new therapeutics and provide a baseline against which disease-modifying therapies can be measured.
"NORD's Natural History Studies project empowers patients and families to help eliminate some of the 'I don't know' in rare disease research, making way for progress," said Peter Saltonstall, CEO of NORD.
"This is good news for the organic acidemia community," said Kimberly Chapman, M.D., a genetic specialist and leading researcher who treats children with organic acidemias at Children's National Health System in Washington, D.C. "I see these wonderful children and families every day and am excited that we are taking an important step to improve their health outcomes."
HemoShear Therapeutics, LLC, which is developing drugs for propionic and methylmalonic acidemias, is supporting the staff needed to initiate the natural history studies, coordinate outreach to patients' families, and collect new data on these devastating diseases.
"We are excited to be on the cutting edge of discovering treatments and possibly cures for these diseases," said Brian Wamhoff, Ph.D., Head of Innovation for HemoShear Therapeutics. "We are working closely with Children's National and the Organic Acidemia Association to take all the steps necessary to be successful."
About HemoShear Therapeutics
HemoShear Therapeutics discovers novel biological targets and advances drug programs to treat metabolic disorders with significant unmet patient need. Our proprietary drug discovery platform, REVEAL-TX™, enables us to create best-in-class, biological human disease models to uncover and explain the underlying mechanisms of disease, translate those discoveries into drug candidates, and predict which drug candidates will treat patients successfully.
Our current drug discovery programs are focused on organic acidemias, a group of rare genetic metabolic disorders, and nonalcoholic steatohepatitis (NASH), a chronic and rapidly growing global disease. HemoShear's collaborators include major pharma companies and leading medical centers.
About Children's National Health System
Children's National Health System, based in Washington, DC, has been serving the nation's children since 1870. Children's National is Magnet® designated, and is consistently ranked among the top pediatric hospitals by U.S. News & World Report. Home to the Children's Research Institute and the Sheikh Zayed Institute for Pediatric Surgical Innovation, Children's National is one of the nation's top NIH-funded pediatric institutions. With a community-based pediatric network, seven regional outpatient centers, an ambulatory surgery center, two emergency rooms, an acute care hospital, and collaborations throughout the region, Children's National is recognized for its expertise and innovation in pediatric care and as an advocate for all children.
About the Organic Acidemia Association
We are a volunteer non-profit organization whose mission is to empower families and health care professional dealing with those afflicted with a family of related metabolic disorders called "organic acidemias". We support early intervention through expanded newborn screening, hold national family/professional conferences to exchange information, and solicit funding to support research leading to improved treatment and the eventual cure of these life-threatening disorders.