From Bench to Bedside: Downloadable Resources

Welcome to our resource centre. Here you will find downloadable guides, educational materials, and direct links to official health authorities across the UK and Europe.

We have gathered these tools to help you, your family, and your healthcare team better understand FOP, navigate NHS treatment pathways, and stay informed about current developments.

All of our downloadable materials are free to access, save, and share with your local doctors, schools, or support networks.

Medical Disclaimer: The information, downloadable materials, and external links provided on this website are for general informational and educational purposes only. They do not constitute medical advice, diagnosis, or treatment recommendations. FOP Friends does not endorse any specific potential treatment or medical intervention. Always consult a qualified healthcare professional regarding any specific medical condition, treatment plan, or health concerns.

External Links Disclaimer: This page contains links to external websites operated by third parties, such as NHS bodies, regulatory agencies, and health technology assessment organisations (including NICE, SMC, AWTTC, DoH NI, NCPE, and the EMA). FOP Friends has no control over the content, updates, privacy policies, or availability of these external sites. Providing these links is intended to assist our community in finding official information, but it does not imply endorsement or responsibility for the content hosted on external domains.

The King’s Fund: Access to new medicines in the English NHS

This King’s Fund explainer outlines how new medicines become available on the NHS in England, tracing the journey from early-stage research and clinical trials through to regulatory safety approval by the MHRA. It details how NICE evaluates clinical and cost-effectiveness to determine if the NHS must fund a treatment, while exploring the budget trade-offs, pricing negotiations, and practical challenges involved in ensuring timely patient access.

Visit: The King’s Fund

Intro to Health Technology Assessments by Beacon

This video explains Health Technology Assessments (HTA) and their application, particularly in the context of rare diseases. It highlights the concept of opportunity cost in healthcare and how HTA aims to ensure fair and consistent decisions on spending by balancing costs against patient benefits.

Watch: Here

PASATRU™ Official Website

PASATRU™ (garetosmab-grts) is a prescription injection manufactured by Regeneron to treat adults living with fibrodysplasia ossificans progressiva (FOP).

While it has received FDA approval for adult patients in the United States, it is not currently licensed or approved for use in the UK.

The official product website provides treatment resources, financial support guidance through the US-based myRARE® programme, and important safety information regarding potential side effects such as skin infections, severe nosebleeds, and pregnancy risks.

Visit: Here

ATEBRIOZ™ Official Website

ATEBRIOZ™ (zilurgisertib) is an oral prescription medicine manufactured by Mirum Pharmaceuticals to reduce new abnormal bone growth (heterotopic ossification) in adults and children aged 12 and older with FOP.

While it has received FDA approval in the United States, it is not currently licensed or approved for use in the UK.

The official website provides patient brochures, treatment navigation through the US-based Mirum Access Plus support programme, and key safety details regarding risks during pregnancy and potential side effects like headaches, joint pain, and nosebleeds.

Visit: Here