“The Soft Bones Scientific Advisory Board was delighted this year to review, once again, highly competitive grants aimed to help patients with HPP,” according to Dr. Michael P. Whyte, Chairman of the Soft Bones Scientific Advisory Board. “The proposal from Dr. Stenhouse will shed light on one of the least understood complications of HPP.”
The proposed study seeks novel insights into the role of the deficient tissue non-specific alkaline phosphatase on lung development and function while exploring the respiratory failure associated with the severe forms of HPP in people. The proposed experiments, using a sheep model, will investigate temporal changes in lung and diaphragm RNA expression and pathology of HPP.
“We are excited to use these funds for this new avenue of HPP research, which will enhance our understanding of the mechanisms underlying respiratory complications in HPP,” says Dr. Stenhouse.
Soft Bones funds research to understand HPP with the goal of finding a cure. The foundation has awarded over $500,000 in research grants since 2014, supporting HPP investigators through this Maher Family Annual Hypophosphatasia Research Grant. More information about HPP as well as existing and ongoing research can be found at www.SoftBones.org.
About Hypophosphatasia: HPP is an inherited, metabolic (chemical) bone disease of remarkably broad-ranging severity that causes life-threatening complications in approximately one per 100,000 births. There can be limb deformity, fractures, and chest deformity that leads to pneumonias. While currently there is no cure for HPP, medical and supportive treatment is directed towards preventing or correcting its signs, symptoms, and complications.
About Soft Bones, Inc.: Soft Bones, the US Hypophosphatasia Foundation, is a patient advocacy group founded in 2009 to educate, empower, and connect patients living with HPP, their families, and caregivers. The Foundation also promotes research of rare bone disease through awareness and fundraising efforts.
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Media Contact
Denise Goodbar, Soft Bones, Inc., 1 973-453-3093, [email protected], www.SoftBones.org
SOURCE Soft Bones, Inc.
