Merritt Engel

  • published FOP Research Strategy in Research 2020-05-28 17:10:27 -0400

    FOP Research Strategy

    IFOPA Research Blueprint 2018-2021

    20% Basic Research

    Answering Priority Research Questions

    The IFOPA will support research that addresses key unanswered questions which will have a near term impact for drug discovery, biomarker identification, and/or for identifying key disease processes in FOP that will lead to a curative treatment.   

    30% Drug Discovery

    Advancing Drug Candidates Towards Clinical Trials

    The IFOPA will support discovery research that expands the development and identification of treatments that alone, or in combination, will prevent or reverse heterotopic ossification and FOP flares.  The IFOPA will build and maintain research infrastructure that reduces the barriers and accelerates drug development

    35% Clinical Trials

    Accelerating Drug Development

    The IFOPA will invest in the development and validation of tools, as well as the analysis of data, that support conducting rigorous clinical trials, requiring the fewest FOP participants and the shortest duration to achieve definitive results

    15% Clinical Care

    Supporting Early Diagnosis and Achieving Optimal Clinical Care

    The IFOPA will advance research efforts to minimize time to diagnosis and to ensure optimal clinical care for those living with FOP

    IFOPA Research Strategy 2018-2021

    Basic Research

    Research Objective: Answering Priority Research Questions

    Research Priority

    1. Establish and maintain a global FOP Biorepository to expand biospecimen access to researchers, enabling the study of FOP biology and testing new treatments

    2. Fund research that delineates the immunologic mechanisms in FOP

    3 Support research that identifies disease mechanisms that will lead to drugable targets for FOP and/or are markers of disease progression

    4. Fund research that further elucidates the mechanisms for muscle biology and regeneration as well as tendon and ligament repair, post bone resection

    5.  Support in research that explores progenitor fate determination into bone (vs muscle) 

    6. Support research that uncovers the genetic and epigenetic modifiers that predict variable phenotypic mosaicism within classic and variant FOP

    7. (Long-term) Establish a genetic database of FOP patients, which is linked to the FOP Registry, with the goal of looking for genetic modifies

    Drug Discovery

    Research Objective: Advancing New Drugs Towards Clinical Trials

    Research Priority

    1. Explore combination treatments that may provide better efficacy, allow for more persistent treatment, or to allow treatment for pediatrics

    2. Fund research that evaluates repurposed drugs that already have a known safety profile and could be a complementary treatment option

    3. Invest in preclinical research that investigates drugs’ effectiveness following post-resection of heterotopic bone

    4. Invest in new therapies that either provide a novel approach to treating FOP or have convincing animal data to warrant further research 

    5. Maintain and ensure unencumbered distribution of FOP mice to test new therapeutic compounds

    6. Promote the replication of promising preclinical; support research that explores the limitations of existing FOP models or the refinement /development of new models

    7. (Long term) invest invalidated research that has the potential to be curative for FOP

    Clinical Trials

    Research Objective: Accelerating Drug Development

    Research Priority

    1. Support the IFOPA FOP Registry and the resulting data analysis to further our clinical understanding of FOP; ensure that all data covered by sharing agreements is transferred into the registry and available for future researchers

    2. Support efforts to identify biomarkers capable of measuring and predicting early disease progression and/or treatment response. Encourage research that validates surrogate endpoints that have utility in clinical trials

    3. Fund research that assesses novel imaging techniques sensitive enough to detect early disease progression

    4. Promote responsible trial design to maximize benefit for the FOP population (e.g. minimizes patient time per benefit gained in each trial)

    5. Develop educational resources on clinical trials to better educate and enable trial enrollment decisions 

    6. (Long-term) Encourage bio-pharmaceutical companies  to identify genetic or other characteristics that  lead to treatment "Responders" from "Non responders“

    Clinical Care

    Research Objective: Answering Priority Research Questions

    Research Priority

    1. Explore non-genetic contributions to phenotypic heterogeneity in FOP, including the role of vaccination, injury, exercise, dietary influences, etc.

    2. Support efforts to educate the international and local medical community on FOP to ensure earlier diagnosis and appropriate management; support efforts to advance pediatric screening for FOP

    3.  Fund translational/clinical research that evaluates optimal pain management strategies for FOP


  • published 2020 Family Gathering in IFOPA News 2020-04-29 14:25:08 -0400

    US Family Gathering to be Held as a Virtual Event

    2020 Virtual Family Gathering Preview

    Read more

  • published Clinical Trials and Studies in PATIENTS & FAMILIES 2020-03-24 13:21:33 -0400

  • published Patient Directory and Registry in Patients and Families 2020-03-18 14:16:12 -0400

    Patient Directory and Registry

    We encourage those living with FOP to join both the FOP Registry and the FOP Directory. 

    The online FOP Patient Directory is available at fopdirectory.org. This helpful resource contains profiles for individuals with FOP, complete with photos and contact information, so you can get in touch with others in the community. The FOP Patient Directory is especially helpful for connecting with people who live near you or people of a similar age. 

    Create your profile for the FOP Directory.

    Frequently Asked Questions about the FOP Directory (Button)  

    Please contact Shelly Oliver, Donor Relations and Administrative Coordinator, if you have additional questions.

    The online FOP Registry is available at fopregistry.org. The registry is a global, voluntary database that collects demographic and disease information directly from individuals with FOP through a secure website. Every person living with FOP has unique information to share. The Registry collects this information to help further research to find treatments and, one day, a cure for FOP.

    To contribute to the registry visit fopregistry.org and select “Join” to register and complete your Enrollment Survey.  

    Learn more about the Registry. 


  • published Meet Our Fundraisers in Fundraise 2020-02-25 11:22:26 -0500


  • published 2019_FOPFamilyGathering 2020-02-10 13:16:35 -0500

  • published 2019_FOPDDF 2020-02-10 13:15:57 -0500


  • published store 2020-01-20 10:56:10 -0500

  • published Research Webinar 2020-01-20 10:54:46 -0500


  • One Family’s Friends Become Friends of the FOP Family

    Read about how two high school friends reconnect to help #cureFOP

    Read more

  • Resilient Living Educational Series

    The Resilient Living Educational Series is an opportunity for members of the FOP community to come together virtually to learn about ways to incorporate habits that support their mental and physical well-being in their day-to-day living.

    Trainings will take place every third or fourth Tuesday of the month at 8 pm EST/7 pm CST/6 pm MST/5 pm PST.

    Each training will be one hour long with the first half consisting of a lecture style presentation of the material and the second half a discussion-based exploration of the exercises associated with practicing that facet of resilience. 

    Trainings will only be available in the English language. 

    Interested in signing up to receive more information about the series and participate in the sessions?                         

    Series Schedule:

    Session 1: What Does Resiliency Mean to You? Recording  Presentation Slides

    We'll start this series off by looking at some of the important aspects of implementing resilient habits and learning why resilience is important for our mind and body.

    Session 2: Emotional Expressiveness Presentation Slides                      

    Emotional expressiveness is the awareness of basic emotions as they occur and the ability to process them appropriately.

    Session 3: Ability to Self-calm Recording  Presentation Slides                                   

    The ability to self-calm is using self-awareness to practice relaxation techniques and self-regulation skills to align our intellect, emotions, actions and words. 

    Session 4: Self-care   Presentation Slides                                                 

    Self-care is choosing healthy behaviors that balance the effects of emotional and physical stressors. 

    Session 5: Hardiness - Tuesday, June 16                                                       

    Hardiness is our ability to place ourselves at the center of the controls and realize that challenges create opportunities for potential learning, growth and personal betterment.

    Session 6: Ability to Self-replenish - Tuesday, July 21                                 

    The ability to self-replenish is recognizing when our inner stores of energy and positive experience are becoming depleted and doing what it takes to fill ourselves up again.

    Session 7: Non-judgement/Self-supporting Talk - Tuesday, August 18     

    Self-supporting talk is challenging unproductive thoughts and replacing critical thoughts with more productive ones. 

    Session 8: Hope and Optimism - Tuesday, September 15                            

    Optimism is remembering that our perception creates reality and that we must have hope to define our goals and make a plan to achieve them.

    Session 9: Sense of Coherence - Tuesday, October 20                                 

    Sense of coherence is focusing on a sense of order and remembering that there are a great deal of resources available in life. During times of distress action eases despair.

    Session 10: Social Support - Tuesday, November 24                                    

    Social support is about turning to the people who support you when you need them and remembering that reciprocating that support is key to a healthy relationship. 

     



  • published Successful Biobank Collection in Florida in IFOPA News 2019-12-20 16:54:15 -0500

    Successful Biobank Collection Event in Florida

    FOP Biobank an invaluable resource for FOP researchers

    Read more

  • published 2019_DDF_recap 2019-12-20 09:09:39 -0500

  • published fda listening session 2019-12-20 09:04:42 -0500

  • published ifopa.org/2019ddf_recap 2019-12-20 09:03:49 -0500

  • Get an Inside Look at the 2019 FOP Drug Development Forum

    Learn more about FOP Talks given by experts from around the world

    Read more


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