Public Policy & Advocacies
Soft Bones works on behalf of the HPP community to increase funding, awareness and necessary access for those with the disease. We are part of the Rare Bone Disease Advocacy Alliance allowing us to lobby for increased federal spending to accelerate research, track prevalence and develop drug treatments and therapies. We have conducted Congressional Briefings and hosted State House events in order to educate our leadership on HPP, rare diseases and the outstanding needs in our community. We have also successfully worked with the Social Security Administration in order to allow those with HPP access to disability benefits.
We work with other organizations with shared interests and have partnerships with multiple groups here in the US as well as worldwide.
As an organization dedicated to promoting research of HPP, Soft Bones raises funds in order to provide grants to further innovative research by new and established investigators in HPP. Since its inception in 2008, Soft Bones has provided a total of $50,000 in research grants.
Research grant recipients include:
- Steven Mumm, Ph.D., Research Associate Professor of Medicine, Washington University School of Medicine and a member of the Scientific Staff at the Shriners Hospital for Children, St. Louis, Missouri. Mumm was awarded $25,000 in [INSERT YEAR] to advance a genetic/outcome study of 144 multi-generation HPP families investigated at Shriners Hospital to help predict the clinical outcome for individuals with specific HPP mutations. The results could guide physicians concerning the prognosis and management of HPP over a patient’s lifetime. Dr. Mumm is a molecular geneticist who studies the DNA of individuals with rare genetic bone diseases, including HPP. He has investigated HPP for 15 years and his laboratory staff has performed TNSALP gene mutation analysis for more than 200 HPP patients and family members.
- Luke Mortensen of Athens, Georgia was granted $25,000 in 2015 to use HPP mice to learn more about the therapeutic potential of transplanting alkaline phosphatase-rich cells into patientsâ€™ bone marrow space. Dr. Mortensen recently joined the Regenerative Bioscience Center (RBC) at The University of Georgia (UGA) where he works closely with the RBC director, Dr. Steven Stice, who is actively working with lab teams on a surgical gel, dubbed “fracture putty” that can heal a broken bone within days. Dr. Mortensen is in the process of opening his own research lab within the RBC, and hopes the seed grant money will provide him the opportunity to gather preliminary data and develop the scientific justification for major external funding. Dr. Mortensen completed a postdoctoral fellowship at Harvard School of Dental Medicine and Massachusetts General Hospital before becoming an Assistant Professor of Regenerative Medicine and Engineering at UGA in 2014. Full press release.
Grants are awarded as one-time seed grants for basic or clinical research directly related to HPP. Proposals are welcome from faculty members at a private research center or university. Applications from senior post-doctoral trainees or research associates are acceptable provided that the individual and his/her mentor have a track record of commitment to research relevant to HPP. This grant cannot be used for investigator salaries or indirect costs. o The Soft Bones Scientific Advisory Board reviews all proposals and those interested should contact email@example.com to request an application.
Rare Disease Awareness Efforts
While HPP is a rare disease, we at Soft Bones know that there is strength in numbers. We have established partnerships with numerous organizations and use our collective voice to increase awareness, lobby for funding, research and policy matters as well as secure more attention and recognition of HPP.
Each year, we participate in activities to commemorate Rare Disease Day (last day of February), we celebrate HPP Awareness Week the last week in October, and we are working with our global partners in an effort to establish October 30 as HPP Awareness Day.
Our partnerships include the National Organization for Rare Disorders (NORD), Global Genes, The National Bone Health Alliance, the Rare Bone Disease Patient Network and the Rare Bone Disease Advocacy Alliance. Soft Bones also works closely with the American Society for Bone and Mineral Research (ASBMR) the premier medical, scientific society in the U.S. dedicated to bone and mineral research.