BOONTON, N.J., Oct. 30, 2019 /PRNewswire/ — To commemorate hypophosphatasia awareness day, Soft Bones, Inc., an organization dedicated to providing information, education and support to those affected by hypophosphatasia (HPP), is pleased to award its tenth annual Maher Family Grant to Dana Gaddy, Ph.D., Professor of Veterinary Integrative Biosciences at Texas A&M University to study a sheep model for hypophosphatasia; and Dobrawa Napierala, Ph.D., Associate Professor of Oral Biology at the Center for Craniofacial Regeneration, University of Pittsburgh School of Dental Medicine; to study adolescents and adults with HPP. In commemoration of its tenth anniversary, Soft Bones is awarding two grants for the first time ever.
Dr. Gaddy, in collaboration with her co-investigators Dr. Sarah White and Dr. Larry Suva, will conduct research using their novel findings of muscle structure and ultrastructural defects in sheep with HPP to determine the cause of muscle weakness commonly seen, but poorly understood, in HPP patients.
“I am inspired to effectively accomplish the proposed research aims with support from this Soft Bones grant,” said Dr. Gaddy. “Our goal is to use the sheep model to identify specific targets of altered muscle function that may be amenable to muscle-specific intervention in patients with HPP. These funds further empower us to resolve muscle weakness in all HPP patients, including my granddaughter,” she continued.
The Scientific Advisory Board for Soft Bones considers grant applications and oversees material development to ensure medical accuracy and provide strategic guidance for research grants. The board, comprised of internationally known, multi-disciplinary experts, is chaired by Michael P. Whyte, M.D., Medical-Scientific Director at the Center for Metabolic Bone Disease and Molecular Research, Shriners Hospital for Children and Professor of Medicine, Pediatrics and Genetics at Washington University School of Medicine in St. Louis, MO.
“A significant appeal of Dr. Gaddy’s proposal is that it moves closer toward the HPP patient,” said Dr. Whyte. “Prior to this, the only animal model was in mice. The sheep model is more ideal for examining bone and dental changes that affect HPP patients and could ultimately lead to therapeutic innovations benefitting patient outcomes,” he added.
The second grant will fund a research project conducted by Dr. Napierala to address the novel role of tissue-nonspecific alkaline phosphatase (TNSALP) in the mineralization process, which is the underlying cause of the majority of skeletal deformities and dental problems in HPP patients.
“My ultimate goal is to understand the molecular bases of human disorders affecting the formation of bone and teeth for the improvement of therapeutic approaches,” said Dr. Napierala. “This grant will allow me to explore the novel, previously unrecognized, function of alkaline phosphatase in bone and tooth cells, as I seek to help identify new treatment approaches that are applicable to various bone and tooth formation diseases.”
“Dr. Napierala’s research aims to provide a better understanding of molecular mechanisms underlying genetic mineralization disorders including rickets, osteomalacia and, most importantly for Soft Bones, HPP,” noted Dr. Whyte. “Her research may provide a premise for evaluating TNSALAP as potential treatment for these disorders.”
These are the tenth and eleventh research grants Soft Bones has awarded since its founding. This year the advocacy group celebrates its tenth anniversary of fulfilling its mission to support HPP patients and families. More information about hypophosphatasia and existing research can be found at www.softbones.org.
Hypophosphatasia is an inherited, ultra-rare, metabolic (chemical) bone disease of broad-ranging severity that causes life-threatening disease in approximately one per 100,000 live births. Depending on the severity of the skeletal disease, symptoms can include deformity of the limbs and chest, pneumonia, and recurrent fractures. While there is currently no cure for hypophosphatasia, treatment is directed towards preventing or correcting the symptoms or complications.
ABOUT SOFT BONES, INC.
Soft Bones was founded in 2009 to provide information and to establish a forum to educate, empower, and connect patients living with hypophosphatasia, their families, and caregivers. The Foundation also promotes research of rare bone disease through awareness and fundraising efforts. Under the leadership of Deborah Nettune Fowler, Soft Bones has raised awareness around the world, including advocating for the disease to receive the policy recognition and research funding it deserves, by bringing attention to children affected by hypophosphatasia around the globe.
Via source, Cison PR Newswire | Two Researchers Awarded Grants to Commemorate Soft Bones 10th Anniversary and Hypophosphatasia Awareness Day
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