Genetic

Medicines for Neurofibromatosis

A group of inherited conditions causing growths (usually non-cancerous) on nerves and skin changes — monitored by specialists, as effects range from mild to more significant.

Education and reference only. This explains which medicines are used and why, in plain language — it deliberately contains no doses and is not a substitute for advice from your doctor or pharmacist. Always discuss your own treatment with a qualified clinician, and check the BNF and the product labelling for prescribing detail.

Quick answer

What is Neurofibromatosis?

Neurofibromatosis refers to a group of inherited conditions in which tumours (usually non-cancerous) grow on or around nerves, and there are often changes in the skin. The most common form, type 1, typically causes flat, coffee-coloured skin patches (café-au-lait spots), freckling in unusual places, and soft lumps on or under the skin (neurofibromas), and can be associated with learning difficulties and, less often, other complications.

  • How it is treated: There is no cure, so care focuses on monitoring, managing symptoms and complications, and support, tailored to the type and the individual.
  • Self-care: Attending regular monitoring, reporting new or changing lumps or symptoms, and accessing learning and developmental support where needed all help.
  • When to seek help: See a GP about multiple coffee-coloured skin patches, unusual freckling, or soft lumps on the skin, especially with a family history, for assessment.

What it is

Neurofibromatosis refers to a group of inherited conditions in which tumours (usually non-cancerous) grow on or around nerves, and there are often changes in the skin. The most common form, type 1, typically causes flat, coffee-coloured skin patches (café-au-lait spots), freckling in unusual places, and soft lumps on or under the skin (neurofibromas), and can be associated with learning difficulties and, less often, other complications. A rarer form, type 2, mainly causes tumours affecting hearing and balance nerves. Severity varies greatly, even within families. It is caused by a gene change and runs in families, though it can also appear without a family history. Diagnosis is based on the pattern of features, examination and sometimes genetic testing.

How it is treated

There is no cure, so care focuses on monitoring, managing symptoms and complications, and support, tailored to the type and the individual. Most people with type 1 have mild disease and need regular review to watch for any complications (such as effects on vision, bones, blood pressure or, rarely, tumours becoming problematic), with treatment as needed. Skin lumps that are troublesome can sometimes be removed. Type 2 needs specialist monitoring of the hearing and balance nerves, with treatment options including surgery, focused radiotherapy and newer medicines. Learning and developmental support is provided where needed. Genetic counselling helps families. Care is usually coordinated by a specialist team.

For this condition, these medicines

Medicine classes used for Neurofibromatosis

Each links to a full, dose-free guide — what it is, how it works, who can and cannot use it, side effects, interactions and FAQs.

Beyond medication

Lifestyle and self-care

Attending regular monitoring, reporting new or changing lumps or symptoms, and accessing learning and developmental support where needed all help. Genetic counselling supports family planning decisions.

When to get help

When to see a doctor

See a GP about multiple coffee-coloured skin patches, unusual freckling, or soft lumps on the skin, especially with a family history, for assessment. Report new or changing lumps, or new neurological symptoms, for specialist review.

999Emergency — call 999 or go to A&E
111Urgent advice — call NHS 111 or use 111 online
GPNon-urgent — see your GP or pharmacist

Not sure how urgent it is? It is always OK to call NHS 111 for advice, day or night.

Answers

Neurofibromatosis: frequently asked questions

Are the growths in neurofibromatosis cancerous?

Usually not — the growths are typically non-cancerous. Occasionally they can cause problems by their size or location, or rarely become cancerous, which is why monitoring is important.

Is neurofibromatosis inherited?

Yes, it usually runs in families, though it can also occur without a family history. Genetic counselling is available to help families understand inheritance and options.

Sources

Where this is drawn from

  • NHS — Neurofibromatosis type 1
  • Nerve Tumours UK / specialist guidance

Related conditions

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