Called upon Mike Penning to move the motion for funding FOP research.

Concurred with Mike Penning's comments about the need for health research but expressed concern over its handling.

I thank the right hon. Member for Hemel Hempstead (Sir Mike Penning) for bringing this debate to the Chamber and for all the work he has been doing to raise these important issues within Parliament. It was good to hear such a fulsome and good description of the condition and how people are affected by FOP. As chair of the all-party parliamentary group on rare, genetic and undiagnosed conditions, I am glad to be able to take part in this debate to highlight the issues facing people with FOP. We have heard from the right hon. Member about just how debilitating this condition is. Usually caused by a gene mutation, FOP is the only known condition where the body changes one organ to another. Bone forms in muscles, tendons and other connective tissues, progressively and irreversibly restricting movement. This severely limits the ability of those affected to perform the most basic tasks of daily life. Children with FOP lose their independence just at the point they should be gaining it. Many of the issues affecting families of children with FOP are experienced by other people across the rare disease communities, including long diagnostic odysseys. The rare disease community has some issues in common, including those long diagnostic odysseys. How long do people have to wait for their condition—I will use that term—to be recognised? There is a lack of clinical awareness with many of these conditions and limited treatment options for far too many people. FOP, as the right hon. Member has said, is perhaps one of the rarest and most disabling of these conditions, with no treatment or cure. Within the rare conditions community, a diagnostic odyssey refers to a common scenario in which delays to accessing the right support and the right treatment—where it exists—can cause irreversible deterioration of an individual’s condition. While there is no treatment for FOP, repeated investigations, such as biopsies, can trigger the condition’s progression. That can be triggered by trauma, too. Furthermore, delayed diagnosis prevents parents from taking action to keep their children safe from situations and activities that could trigger injuries and flare-ups. Unfortunately, a diagnostic odyssey is the norm for many children with FOP. Getting a diagnosis takes one and a half years on average, and more than half of people with FOP get the wrong diagnosis in the first instance, as we have heard. Despite genetic tests being available, FOP is not included in the national genomic test directory for rare and inherited conditions. Can the Minister explain why it is not included in that directory? What plans do the Government have to change that position? The real hope for FOP, as we have heard, is new research. Like much of the research into rare conditions, it is likely to have far-reaching benefits for more common illnesses, such as osteoporosis, childhood brain cancer and heart disease. At the moment, the Government are overseeing a decline in research and international life science competitiveness, with commercial clinical trial activity in the NHS declining over recent years. We have heard from the right hon. Member for Hemel Hempstead about the STOPFOP trial, which is supported by funding from the European Union’s Innovative Medicines Initiative as part of Horizon 2020. Is it not ironic that we are discussing this on the day we have heard that we are now in the Horizon programme? Thank goodness we are; it is an important move. However, there have been two years of wasted opportunities and uncertainty for people going through trials and research, such as people with FOP. I understand that researchers would have to apply for new funding from the scheme to carry on with the STOPFOP trial. How will the Government ensure that funding continues to be available to allow the trials to continue, and to ensure real progress in diagnosing and treating FOP? As the right hon. Member for Hemel Hempstead said, time really is of the essence if we are not to lose the benefit of the work already done and if we are to give those with FOP, and those who may be born with the condition in future, the best chance of the earliest possible diagnosis and treatment.

I thank the contributors for their input and express my interest in rare diseases, particularly FOP. I highlight the rarity of FOP with approximately 70 known cases in the UK and two confirmed cases in Northern Ireland involving twin sisters. Emphasising early diagnosis as key to improving patient outcomes, Jim calls on the House to support additional funding for the STOPFOP trial and research into Saracatinib as a potential treatment. He also underscores the importance of raising awareness among healthcare professionals about FOP, advocating for broader diagnostic measures including neonatal screening.

Congratulates the right hon. Member for Hemel Hempstead on securing the debate; highlights the importance of rare diseases being collectively widespread and the need for highly specialised treatment; acknowledges the UK Rare Diseases Framework and its key priorities for the next five years; praises the Scottish Government's commitment to implementing their Rare Disease Action Plan with four key themes: faster diagnosis, increased awareness among healthcare professionals, better care coordination, and improved access to specialist treatment and drugs; discusses the rapid development of genomic medicine and its impact on patient treatment; commends the Scottish Government’s genomics strategy and their allocation of a revised budget for genomics in 2022-23; emphasises the need for more funding to support research and transformative changes in genomics over the next five years.

The debate focuses on Fibrodysplasia Ossificans Progressiva (FOP), an ultra-rare condition affecting one person in a million. Early diagnosis is crucial for managing the progression of FOP, as 52% of people with FOP receive an incorrect initial diagnosis leading to delays in care. Genetic testing can confirm FOP and should be included in the national genomic test directory for rare diseases. The debate also highlights the importance of newborn screening for FOP and digital education resources for clinicians. Early signs include under-turned big toes, and families need better access to information and guidance on managing the condition. Care for individuals with FOP is complex and specialist, necessitating a workforce plan that increases the number of care workers to support those living with rare diseases. Research into FOP holds promise for treating more common illnesses like osteoporosis, childhood brain cancer, and heart disease.

In an intervention, Mike Penning highlighted the devastating effects of limb amputations in cases of FOP. He explained that amputations are often counterproductive as they cause further damage to muscles which can turn into bone.

Acknowledged the importance of FOP, thanked Sir Mike Penning for securing the debate, expressed admiration for families affected by the condition and their efforts in raising awareness and funding research. He committed to meeting with the Science Minister on this issue. Emphasised the Government's commitment to rare disease research through significant funding, highlighted the progress made in newborn screening, and discussed opportunities under Horizon Europe for further research.

Welcomed to her position on the Front Bench. Mentioned concerns about the National Screening Committee's decision-making process regarding new conditions and newborn screening for FOP.

Asked specific questions regarding research into FOP, highlighted the importance of centres in Newcastle that are funded by the UK rare disease funding. Raised concerns about the complexity of bidding processes and time taken to receive decisions.

Participated in discussions highlighting the need for continued research, including international collaboration and newborn screening advancements.

Mr Penning expressed gratitude for the debate and discussed the importance of early screening to save NHS money. He mentioned that only one in 1 million cases are known currently, highlighting the need for more active engagement with the condition to attract specialist consultants and scientists who can work on this rare disease. Mr Penning also noted a moving moment outside No. 10 Downing Street where affected children handed a letter to the Prime Minister's office.