Putting you in the heart of the rare community – Childhood Tumour Trust (CTT)
Estimated reading time: 4 minutes
Patient advocacy groups deliver their top three unmet needs and main asks of industry to best serve their rare community. This week’s insights come from Vanessa Martin, CEO, Childhood Tumour Trust (CTT)
The Childhood Tumour Trust (CTT) is a UK charity dedicated to supporting children, young people and their families affected by neurofibromatosis Type 1 (NF1) — a complex genetic condition that can cause tumours to grow along nerves and can lead to a range of health, learning and social challenges. We provide opportunities for connection through camps, days out, online activities and youth programmes, helping those with NF1 feel less isolated and more empowered. CTT also offers educational resources for families, schools and healthcare professionals to improve understanding and ensure better support. We raise awareness of NF1 in the wider community, advocate for improved care and early diagnosis and participate in research to improve future outcomes. Our mission is to ensure no child, young person or carer affected by NF1 faces their journey alone, while driving positive change in awareness, support and treatment.
Neurofibromatosis Type 1 (NF1) is a common genetic condition affecting around 1 in 2,500 people. NF1 can affect anyone and can cause tumours to grow on nerves anywhere in the body, as well as increasing the risk of certain cancers and brain tumours. It may lead to complications such as skin changes (café-au-lait spots and freckles in unusual places), benign lumps under or on the skin (neurofibromas), learning difficulties, bone abnormalities, vision problems, headaches and high blood pressure. Symptoms and severity vary greatly between individuals. Early diagnosis and regular monitoring help manage health, wellbeing and potential complications.
What are your top three unmet needs or main asks of industry to best serve your rare ecosystem?
NF1 is often missed or misdiagnosed in childhood because symptoms can be subtle or mistaken for other conditions. Industry could help by developing simple, practical screening tools and targeted education materials for healthcare professionals. Earlier diagnosis allows for timely monitoring, access to educational support and cancer screening where needed—improving long-term outcomes.
NF1 affects more than physical health—it impacts learning, mental wellbeing and quality of life. Industry could partner with charities to develop digital health tools, mental health support programmes and coordinated care platforms that address the full spectrum of needs. This would help bridge the gap between medical treatment and everyday living with NF1.
NF1 is a complex, progressive condition that requires lifelong monitoring. Many people with NF1—particularly those over the age of 18—are lost in the system and do not receive the support they need. Industry could help by promoting standardised care guidelines, providing accessible resources for GPs and other healthcare professionals, and creating clear information for teachers. This would ensure consistent monitoring, better understanding in schools,and improved outcomes for individuals throughout their lives.
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