FDF launches the FD/MAS Patient Registry
FDF and NORD launched a cutting-edge study of fibrous dysplasia/McCune-Albright syndrome on October 31, 2016. The new study, the FD/MAS Patient Registry, creates a platform for patients around the world to share information about FD/MAS. Its purpose is to build an international resource to be used by scientists in future research. Within 6 months, 450 patients had joined, making this the largest study of FD/MAS in the world.
Grant Awarded to Researcher via the Million Dollar Bike Ride and UPenn Medicine
We’re thrilled to share that the 2016 awardee is Dr. Mara Riminucci of Sapienza University of Rome. The award is highly competitive and provides funds for a research proposal to investigate fibrous dysplasia and McCune-Albright syndrome (FD/MAS). Thanks to Team Captain Cindi Brandt Levin, this year the FD/MAS community raised $74,000 to support cutting edge research that may lead to effective treatments for FD/MAS patients.
Study Publishes Findings on Effectiveness of Bone Grafting Surgeries
A study in the Journal of Bone and Joint Surgery establishes bone-grafting appears to have limited value in treating fibrous dysplasia and McCune-Albright syndrome. . This study was partially supported by a grant from the Fibrous Dysplasia Foundation. Read the full article.
FDF Supports a Tier II Pipeline-to-Proposal Project
The Patient Centered Outcomes Research Initiative continued to support the FDF supported project to advance comparative effectiveness research (CER) of FD/MAS. This is the second step in a three tiered process of designing and launching a patient-centered research study. During Tier II, the project solicited community input on potential research questions, and will built partnerships with researchers interested in pursuing those questions.
Grants Awarded to Two FD/MAS Researchers via the Million Dollar Bike Ride
Through the efforts of Team FD and the Penn Medicine Orphan Disease Center, two grants of $58,500 will go to qualified and esteemed researchers, with exciting ideas that bring the FD/MAS community closer to effective treatments.
The winning proposals are:
Mouse Models of Fibrous Dysplasia as a Tool for Developing Rational and Effective Therapies, led by primary investigator Dr. Mara Riminucci of Sapienza University of Rome,
Development of Next-Gen Animal Models for Fibrous Dysplasia(FD)/McCune-Albright Syndrome(MAS), led by primary investigator Dr. J. Silvio Gutkind of Moores Cancer Center, UC San Diego.
Read more about the progress of these studies.
FDF Supports a Tier I Pipeline-to-Proposal Project
The Patient Centered Outcomes Research Initiative supported the FDF and FD/MAS patients as they pursued a comparative effectiveness researcher (CER) project. The project, Bridging Rare Disease Patients and Data through Novel Research Partnerships, is committed to building an online hub to support patients and the community that serves them with tools and relevant information to support CER in the future. During this Tier, the project will seek patient, researcher, and clinician in put on the research questions that are most important to them and most urgent to study.
The FDF was selected for a NIH pilot program
The Office of Rare Diseases Research (ORDR), National Center for Advancing Translational Sciences (NCATS), NIH has launched a pilot program to establish a Global Rare Diseases Patient Registry and Data Repository (GRDR). The Fibrous Dysplasia is one of 15 organizations chosen to participate before a patient registry was established.
Grant Awarded to FD/MAS Researcher
Following consultation with the FDF Scientific Advisory Council, the FDF Board of Directors awarded funding to Harvard Medical School/ Massachusetts General Hospital researcher Murat Bastepe, MD, PhD, to breed and analyze a mouse model of FD/MAS. Mouse models are critical research tools that allow researchers to investigate disease pathogenesis and test novel treatments.
Scientific Conference Held in Bethesda, MD
FDF and an unrestricted grant from Zimmer funded a FD/MAS Scientific Conference held at National Institutes of Health in Bethesda, MD. Many FD, MAS and cherubism researchers and clinicians came together to share information.
Front Row (Left to Right): Charles Harles, President FD Foundation, Marilyn Kelly, RN, MS, Pam Robey, Ph.D, Maria Papakaki, DMD, MD, Allison Boyce, MD, Lee Weinstein, MD
Row 2 (Left to Right): Sunday Akintoye, BDS, DDS, MS,Dempsey Springfield, MD, Mara Riminucci, MD, PhD, Erica Eugster, MD,Natasha Cherman, PhD, Beth Brillante, RN, MBA, Rachel Ganfi, MD, Deborah Gensburger, MD, Khalda Ibrahim, BS
Row 3(Left to Right): Edward Hsiao, MD, PhD, Lawrence Lustig, MD, Murat Bastepe, MD, PhD, Nisan Bhattacharyya, PhD, Mahesh Mankani, MD, Patrick Mantyh, PhD, JD
Row 3A (Left to Right): Arabella Leet, MD, Jeff Kim, MD, Leonard Kaban, DMD, MD, William Chong, MD
Row 4 (Left to Right):Yu Ray Chen, MD, Janice Lee, MD, DDS, MS, Roland Chapurlat, MD, PhD
Row 5 (Left to Right): Robin Pals Rylaarsdam, PhD, Robert Stanton, MD, Lynn Lindaman, MD, Sergei Kutznetsov, PhD
Row 6 (Left to Right): Michael Levine, MD, Ernesto Ippolito. MD, John Northup, PhD, Shlomo Weintraub, MD
Row 7 (Left to Right): Michael Collins, MD, Paolo Bianco, MD, Kenn Holmbeck, PhD
Row 8 (Left to Right): Scott Paul., MD, Fred Singer, MD
Row 9: Ernst Reichenberger, PhD
Row 10: Edmond Fitzgibbons, MD