FOP Friends Blog

The FOP Patient Connection Registry 

The FOP patient’s registry is an exciting initiative developed by the International Fibrodysplasia Ossificans Progressiva Association (IFOPA) to help understand and learn more about FOP. This research study will collect and report selected medical information on individuals living with FOP, through a series of online surveys. 

“A registry is essential for understanding any rare disease and developing potential therapies.” Said Betsy Bogard, IFOPA Director of Global Research Development, talking about the benefits of an FOP patient Registry. “One of the biggest challenges in very rare diseases such as FOP is limited patient data. The FOP Connection Registry puts the power of data collection directly into the hands of the patient community and will allow us to bring together a much larger volume of data.”

Once registered, participants will be able to complete as much of the surveys as they would like - the more information the better, but if participants can only fill in a few survey items, even that data will be helpful. The surveys may be completed by the individual living with FOP or by someone else – for example, a parent, relative, or other caregiver on the participant's behalf. In some cases, more than one person will complete the survey questions – whatever method works best for the individual and family will work for the Registry.

Not only will the registry help increase knowledge of FOP, aid research, and help guide clinical trial design, it will critically show pharmaceutical companies that the FOP community are pro-active in the search for a cure. Together we will make a difference!

For more information on the registry and how to join visit:

Clementia Pharmaceuticals are currently trialling a treatment for FOP.  Want to learn more? 

Clementia have produced a document, with UK information, for patients who want to find out more. Including the answer to; Who is Clementia? What are the objectives of Clementia's clinical trial program? Who is eligible?...and more.

 Click HERE or on the link below to download the document.

Mari Wyn Jones
Tuesday, 26 January, 2016 - 09:51

FOP Friends are delighted to announce that we have successfully applied for a grant from The Big Lottery Fund, Awards for All England programme to help fund the FOP Friends UK Conference & Family Gathering, May 2016.

The first UK FOP Symposium for FOP patients and their families held in May 2014 was a resounding success and we are delighted to have the support of The Big Lottery Fund for the 2016 conference. FOP Friends are busy planning for this event which will include presentations, clinical appointments, workshops and advice for people living with FOP. The event will take place in Manchester, UK at the Radisson Blu Airport Hotel. The main event is on Saturday 21st May 2016 with pre-booked clinical appointments available with our world renowned FOP experts on Friday 20th, Saturday 21st (afternoon), and Sunday 22nd.

FOP experts from around the world will be in attendance with clinical sessions being led by Professor Fred Kaplan and Dr Robert Pignolo (University of Pennsylvania, USA) and Dr Richard Keen (Royal Stanmore Hospital, UK). Workshops will be held during the day with an opportunity to break off into groups to discuss topics of interest.  Childcare and activities will be provided for those with young families.

Booking information for this event, including clinical appointments and childcare will be announced soon!

You can see some information from the 2014 Conference & Family Gathering HERE.

Lottery logo


Mari Wyn Jones
Thursday, 14 January, 2016 - 11:59

The year has seen some exciting breakthroughs in the search for new treatments for FOP. We’ve known for some time now that the underlying cause of FOP is a single faulty gene called ACVR1. This gene produces an overactive receptor protein which acts as a critical messenger for growth factors that stimulate bone formation. In FOP, the mouth of the receptor loses proper control and sends out too much signal that says ‘make more bone, make more bone, make more bone’. Fortunately, there are many ways researchers could try to correct this faulty activity and stop the unwanted bone formation.

The team in Oxford have been taking lessons from the decades of cancer research. Just like the bone in FOP, many tumours grow because of the excessive growth factor signals that are sent from the mutated receptors found on cancer cells. Big pharmaceutical companies have developed lots of drug molecules that bind to the mouths of these receptors (just like babies’ dummies) to block their messages. The team in Oxford searched through libraries of these drug molecules to see if any of them could also bind to the mouth of the FOP receptor. Excitingly, they discovered that one of these clinical molecules was extremely good at stopping the FOP signals too. This compound has advanced to late stage clinical trials for various human cancers.  Encouragingly, it meets all the criteria the team have been striving to achieve: it is potent, selective, stable and safe to use.

Following the discovery of the compound, the team performed a number of additional experiments to validate the suitability of the compound for clinical trials in FOP. This has included collaborating with a colleague Dr. Yu in Boston to test it in FOP mice. After a nervous wait, the team were delighted to hear that the compound had successfully prevented the mice from developing the bone lesions typical of FOP. A hopeful and incredible discovery! The team are now preparing grant applications to the government to get funding to support a clinical trial in FOP late next year. Note, this is a different research path to the one being trialled by Clementia who are using an experimental drug called palovarotene that acts elsewhere to stop the path of building cartilage and bone.

Thank you!

The team look forward to very exciting times ahead and would like to take this opportunity to thank everyone for their generous, ongoing support.



Mari Wyn Jones
Wednesday, 30 December, 2015 - 12:35

Doing your Christmas shopping? Donate when shopping online- its easy to set up and it costs you nothing! 

FOP Friends is registered with who help us to raise money when our supporters (you) shop online. You can shop with over 2,700 well known stores and each will donate up to 15% of what you spend. It does not cost you anything, you pay the same price as everyone else. The only difference is that your purchase also raises money for FOP Friends. 

Mari Wyn Jones
Wednesday, 30 December, 2015 - 12:32

We are excited to introduce FOP Friends' first employees: David Pilkington and Mari Jones.

Funded for 12 months thanks to the support of GDUK, along with other minor grants, their combined skills will make a formidable team to help the charity grow.

Until now the charity has been run solely by the charity trustees on a volunteer basis and we hope the addition of David and Mari will help us meet our annual fundraising targets and reduce some of the volunteer workload. 

David Pilkington started working as our new Grants & Trusts Manager in August. David is a graduate from the University of Dundee with a degree in Philosophy and Economics. His career has included appointments in companies and public bodies.

In the charitable sector he has experience of acting as Chair and Trustee with particular responsibilities for strategic planning and fundraising through charitable trusts and HMG sponsored organisations and departments. Since joining FOP Friends he’s been amazed and impressed by the commitment and imagination of our FOP families and their supporters towards fundraising and the dedication of both families and clinicians to finding a  cure for FOP.

He says that he is determined not to let the side down and keep up a steady stream of hopefully persuasive applications to help raise awareness of FOP and sustain our ability to fund FOP research every year.

David is married with four children. He lives in Altrincham just a short drive to the FOP Friends office or should that be a bike ride? The possibility is a work in progress, he says.

My name is Mari Jones and in August I started working as FOP Friends Fundraising and Medical Liaison Officer. Having graduated with a degree in Genetics in 2014, people might expect that I would know about Fibrodysplasia Ossificans Progressiva (FOP). In reality, like many others, I had not even heard of FOP. Affecting 1 in 2 million people worldwide, FOP is one of the rarest genetic conditions known to medicine. Raising awareness of FOP with medical professionals and with the public will therefore be a critical part of my role and I will also provide support for FOP Friends fundraisers.

I have experience working in the pharmaceutical industry and at university laboratories researching drug targets for diseases, with similar approaches to FOP research. My previous charity involvement includes volunteering at the Royal Manchester Children’s Hospital and with the elderly at a community centre. In my spare time I enjoy hiking, singing and spending time at my parent’s farm in Wales.

When I first came across FOP Friends I was blown away by how far the charity had come since establishment in 2012 and am committed to working with the FOP Friends trustees to ensure the charities future development and success. 
Mari Wyn Jones
Wednesday, 4 November, 2015 - 11:31


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