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Hemoshear Part of Group Receiving NORD Grant

Thursday, April 21, 2016  
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The Organic Acidemia Association, together with HemoShear Therapeutics and Children's National Medical Center, has received 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, 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."

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