Merritt Engel

  • Meet the 2019 ACT for FOP Grant Awardees

    IFOPA Announces Record-Breaking ACT (Accelerating Cures and Treatments) for FOP Grant Funding

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  • published In Honor of Natalie McGuire 2019-12-04 11:25:10 -0500

  • published In Honor of Ashley Martucci 2019-12-03 13:49:00 -0500

  • published FOP Biobank in Research Tools 2019-11-26 16:01:56 -0500

    FOP Biobank

    The International FOP Association (IFOPA) maintains a FOP biobank on behalf of the FOP research community. Donated samples from people with FOP, and from control donors, are stored in the Biobank at Vanderbilt University Medical Center for redistribution and use by academic and industry researchers. It is the IFOPA’s goal to place these samples into the labs of qualified researchers who will further our knowledge about FOP and accelerate the development of new treatments. 

    Available Samples 

    View the list of available samples. 
    Samples are being added to the FOP Biobank on a continual basis. Please check back to view the latest available samples.

    The Acquisition Process 

    To acquire biospecimens from the FOP Biobank, please complete a FOP Biobank Request Form. The Request Form will be reviewed by a Scientific Review Committee for scientific merit, research impact, feasibility and availability of appropriate biosamples.
    Approved applicants will be sent a Uniform Biological Material Transfer Agreement (UBMTA) that must be signed by the principal investigator (PI) and receiving institution prior to sample release. The PI is responsible for obtaining any necessary Institutional Review Board (IRB) approvals or waivers, as required by their institution. 
    Once all documents are received by the IFOPA, the FOP Biobank will be notified of the aliquots to be shipped and will communicate directly with the PI to coordinate delivery. As part of the UBMTA, investigators will be required to provide a final scientific report or a publication to the IFOPA and notify the IFOPA of any additional publications.

    Partnering Opportunities 

    The IFOPA welcomes the opportunity to partner with sponsors of clinical trials or research studies to collect biosamples and donate the materials to the FOP Biobank. These biosamples will be made available for access to qualified researchers and distribution governed through a Scientific Review Committee. Please contact Adam Sherman if you are interested in partnering with the IFOPA. 

    Acknowledgments & Sponsorship 

    The IFOPA oversees the FOP Biobank on behalf of the FOP research community and thanks to generous support from Blueprint Medicines, Clementia, an Ipsen company, and Regeneron Pharmaceuticals.
    We thank the invaluable in-kind support that has been provided by Margo Black (Vanderbilt University) and Drs. Charles Hong (University of Maryland), Ed Hsiao (University of California, San Francisco), Dan Perrien (Emory University), Bob Pignolo (Mayo Clinic), Eileen Shore (University of Pennsylvania), and Yan Ru Su (Vanderbilt University).  

  • Save the Date for A Transatlantic Burns Supper Fundraiser

    Special fundraiser comes to the United States for the first time

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  • FOP Drug Development Forum and Family Gathering Come Together in Orlando

    A Special Week of Meetings Further Unites the FOP Community 

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  • published News from BioCryst Pharmaceuticals in FOP News 2019-11-22 10:53:46 -0500

    Exciting News from BioCryst Pharmaceuticals

    BioCryst launches Phase 1 trial for FOP

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  • published Ability Toolbox Program in Programs & Services 2019-11-20 12:28:23 -0500

    Ability Toolbox Program

    The IFOPA is excited to add the Ability Toolbox Program to the list of Family Service programs in 2019. The focus of this program is to empower individuals with FOP by promoting independence through the use of tools and home adaptations.   

    Ways the Ability Toolbox Program Can Support You

    INCREASING opportunities for independence and promoting higher levels of confidence.

    ASSISTING community members with developing sustainable daily habits that promote decreased reliance on caregivers, and balance in the lives of families affected by FOP.

    EMPOWERING people to solve problems by creating tools designed to meet their own unique needs.

    Watch the Ability Toolbox Presentation at the 2019 Family Gathering

    We’ll soon be posting the Ability Toolbox Presentation from the 2019 Family Gathering in Orlando, Florida. Hear the FOP community member panelists discuss tools they use every day to maintain their independence during daily routines. See examples of a few tools from these different categories: Eating and Cooking, Toileting, Bathing/Showering, Dressing and Grooming, School and Workplace, Recreation and Hobbies, Medical and Preventative Tools, and Adaptations Around the Home

    **The webinar was recorded live in English but can be viewed with translated subtitles

    Get involved in the Ability Toolbox Program

    Watch for our weekly posts on social media to see featured tools and tips for staying independent during daily routines.


    1. Watch videos of community members demonstrating various tools on our YouTube channel.
    2. Learn from our larger FOP community as they share their feedback and recommendations at small in-person meetings and larger Family Gathering presentations in the US and around the world.
    3. Speak with the Family Services Coordinator, or one of our Tool Ambassadors, to have your questions about tool integration and home adaptations answered. Call +1 (913) 558-1879 or email Karen Kirchhoff, Family Services Coordinator, to learn more about the Ability Toolbox program.

    Coming Soon! 

    A New Online Guidebook!

    We're creating a new Online Guidebook featuring both manufactured tools, as well as homemade tools submitted by FOP community members. The Guidebook will be continuously updated with the latest tools that FOP community members find useful in their day-to-day lives.

    Do you have a tool that you would like to share with the FOP Community? Email Karen Kirchhoff family services coordinator.

    The Ability Toolbox program is made possible by grants from The Radiant Hope Foundation, Ipsen, Regeneron Pharmaceuticals, and Blueprint Medicines.

  • published A Birthday Wish Come True in IFOPA News 2019-11-19 12:01:42 -0500

    A Birthday Wish Comes True

    AJ’s Lemonade Stand Raises Over $6,600

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  • An Important Announcement from Blueprint Medicines & Ipsen

    Ipsen and Blueprint enter exclusive agreement in FOP

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  • published ZipperQ8 Crosses $1 Million Mark! in IFOPA News 2019-10-29 15:40:53 -0400

    ZipperQ8 Crosses $1 Million Mark!

    Success of Beloved Annual Event is Inspiration for Other FOP Fundraisers

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  • IFOPA Announces Record-Breaking ACT (Accelerating Cures and Treatments) for FOP Grant Funding

    The IFOPA offers the ACT (Accelerating Cures and Treatments) for FOP grant program to accelerate the development of new drugs for FOP. The research grant program provides, through a competitive application process, funding to scientists conducting research on fibrodysplasia ossificans progressiva (FOP).

    2019 Funders

    The ACT for FOP Grant Program was launched in 2015 by friends and family of Sona Brinkman. Today others who are passionate about curing FOP have joined them in funding the ACT for FOP Grant Program with lead gifts from: Joshua’s Future of Promises, Canadian FOP Network, FOP Australia, FOP Friends®, FOP Italia and friends and family of Sona Brinkman. The IFOPA's 2019 #GivingTuesday Campaign on Tuesday, December 3 will raise additional funds for the ACT for FOP Grant Program.

    2019 Grant Awardees

    Development of Humanized Antibodies to Mutant ACVR1 and Next-Generation Bispecific Anti-ACVR1-inflamatory Pathway Inhibitors

    Principal Investigator: Nicholas C Nicolaides, PhD
    Institution: Navrogen
    Country: United States
    Award Amount: $90,000

    Project Description: The research team at Navrogen has developed a novel approach to treating FOP by developing immune-based agents that target the FOP receptor and an immune component believed to contribute to the underlying cause of FOP flare ups.  One of the benefits of using this approach in FOP is the preferential targeting of the mutant FOP receptor, thus enabling the normal receptor to perform its natural functions.   

    Recently, the Navrogen team has shown that this approach was able to significantly impact heterotopic ossification (HO) in a FOP mouse model.  Funds from the ACT for FOP grant will enable the further development and testing of these agents for advancement towards human testing.

    In Vivo Evaluation of Antisense Oligonucleotide Therapy for FOP in Humanized Model Mice

    Principal Investigator: Toshifumi Yokota, PhD
    Institution: University of Alberta
    Country: Canada

    Award Amount: $60,000

    Project Description: 

    The University of Alberta team led by Dr. Yokota is looking to continue their research in FOP using antisense oligonucleotides (AONs) as way to stop FOP.  This approach is unique in that the AONs target the mutated gene products (i.e. mutated ACVR1/ALK2 receptor) in FOP. As a result of prior funding from the IFOPA and the Canadian FOP Network, the University of Alberta has developed compounds (called a “gapmers”) that target the mutated part of the ACVR1/ALK2 gene.  These gapmers were able to efficiently reduce most of the mutated gene products, while allowing normal (healthy) receptors to remain unaffected. This approach has been proven effective in other rare diseases, including familial hypercholesterolemia and hereditary transthyretin amyloidosis. 

    New funding from the ACT for FOP grant program would allow Dr. Yokota’s team to optimize these gapmers to better differentiate between normal and mutant gene expression and to better understand the dosing and delivery of these investigational drugs.

    Neutrophil Based Translational Therapies for Fibrodysplasia Ossificans Progressiva

    Principal Investigators: Amanda Huber, PhD & Benjamin Levi, MD
    Institution: University of Michigan
    Country: United States
    Award Amount: $65,000

    Drs. Amanda Huber and Benjamin Levi at the University of Michigan are investigating the role of the innate immune system in FOP.  This research team leverages the expertise of Dr. Huber who is an immunologist by training and Dr. Levi who is a stem cell biologist with expertise in the response to injury. They will build on their exciting preliminary data identifying the central role of neutrophils and their function in FOP. Drs. Levi and Huber have identified the pathway through which this alteration in neutrophil function occurs which they propose to target with two new pharmacotherapies. 

    Funding of this research will allow these investigators to further our understanding about the inflammatory response in FOP and would potentially identify two new inhibitors (one which is currently regulatory approved) that could help mitigate HO formation for people living with FOP. 

    PI3K inhibitors as a New Therapy for Fibrodysplasia Ossificans Progressiva

    Principal Investigator: Francesc Ventura Pujol, PhD
    Institution: University of Barcelona
    Country: Spain
    Award Amount: $60,000

    Project Description: Investigators at the University of Barcelona, let by Dr. Francesc Ventura, are looking to continue their research reported in Cell Signaling and Biology. Dr. Ventura described the ability of an inhibitor, BYL719, to block the formation of heterotopic bone in FOP mice.  BYL719 works by inhibiting phosphoinositide 3-kinase (PI3Kα), an enzyme that is involved in many cellular functions including cell growth, proliferation, and differentiation.  

    Research conducted under the ACT for FOP grant program will analyze the mechanisms of BYL719 inhibition of heterotopic ossification (HO) in FOP mice; including which cell types are involved, which steps of HO are inhibited and optimize the timing of BYL719 administration. This research could lead to a new way of treating of people with FOP.

  • Cars2CureFOP Turns a Vehicle Into Support for FOP Families

    Donate Your Vehicle to Help Support the FOP Community 

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  • published FOP Holiday Ornaments 2019-10-02 15:44:41 -0400

  • published ASBMR 2019 Presentations in FOP News 2019-09-20 14:53:54 -0400

    ASBMR 2019 Presentations

    11 FOP Presentations Will Be Made At The Annual Meeting

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  • published Holiday Ornaments 2019 2019-09-20 12:19:05 -0400

  • Million Dollar Bike Ride Grant Program

    September 23 is the Deadline to Apply for Two FOP Research Grants

    The IFOPA is pleased to partner again this year with the Orphan Disease Center (ODC) at the University of Pennsylvania to expand FOP research grants through the 2019 Million Dollar Bike Ride Pilot Grant Program

    Two FOP research grants valued at $40,208 each are now seeking applicants. The Orphan Disease Center’s application process is two steps – a Letter of Interest (LOI) due at 8 pm EST on September 23, 2019, followed by an invitation to complete a full application. This Request for Applications (RFA) is open to the international community. All individuals holding a faculty‐level appointment at an academic institution or a senior scientific position at a nonprofit institution or foundation are eligible to respond to this RFA. The two areas of FOP research focus for grant consideration are:

    1. Research that seeks to identify biomarkers, including novel imaging techniques, capable of measuring and predicting early FOP disease progression and/or treatment response.
    2. Research that investigates and further elucidates the immunologic mechanism in FOP.
    Awardees of the research funding may have access to the IFOPA’s FOP Mouse Model (IFOPA will support the cost of animal models with the exception of shipping) or available samples from the IFOPA's FOP Biobank, if needed. Please contact the IFOPA at for further details on these resources.

    These grants are made possible by Team #cureFOP riders who participated in the Million Dollar Bike Ride and the IFOPA.

    Review all program details and application instructions

    Official Program Announcement From the Orphan Disease Center

    The Orphan Disease Center (ODC) at the University of Pennsylvania is pleased to announce the 2019 Million Dollar Bike Ride Pilot Grant Program. The program is now open and offering up to 38 different research grant opportunities focusing on 30 different rare diseases. This program provides a one‐year grant to support research related to a rare disease represented in the 2019 Million Dollar Bike Ride. Number of awards and dollar amounts vary per disease based on fundraising totals by each disease team. This Request for Applications (RFA) is open to the international community. All individuals holding a faculty‐level appointment at an academic institution or a senior scientific position at a nonprofit institution or foundation are eligible to respond to this RFA. We ask that you please share this funding opportunity with your department or other relevant researchers. 

    For more details about this grant program, rare disease focus areas, and how to apply, please visit the Orphan Disease Center. The RFA Guidelines are available. Letters of Interest (LOIs) are due no later than Monday, September 23, 2019 by 8 pm EST. Please refer to our website for instructions on LOI submission.  

    Please contact Samantha Charleston by email, or call +1 215-573-6822 with any questions.

  • published The Miller Family Story in IFOPA News 2019-09-16 14:52:24 -0400

    The Miller Family Story

    A Life of Devotion. A Life of Determination.

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  • Semi Good Lookin’ Wins Over $20,000 for the IFOPA

    Battle of the Biotech Bands Check Presentation

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