The Think Again. Think NP-C campaign is dedicated to supporting early diagnosis of Niemann-Pick type C disease (NP-C) to ensure patients can access treatment and support.

About the Think Again. Think NP-C campaign

What is the Think Again. Think NP-C campaign?

Think Again. Think NP-C is an international campaign, which aims to support healthcare professionals unfamiliar with Niemann-Pick type C disease (NP-C) to recognise the key signs and symptoms of NP-C. This will help patients by speeding up diagnosis to ultimately optimise care and treatment outcomes.

Why is the Think Again. Think NP-C campaign needed?

NP-C is commonly undetected or misdiagnosed. This is often due to its highly variable clinical presentation characterised by a wide range of symptoms that individually are not specific to the disease.1-3

Due to its challenging presentation the journey to diagnosis of NP-C can be long and frustrating for patients and their families.4 The average delay in diagnosis is five to six years from onset of neurological symptoms.5 This campaign aims to reduce the length of time to diagnosis by supporting healthcare professionals to recognise the symptoms of NP-C in order to be able to Think NP-C.

Early diagnosis means patients can access support, which can improve their quality of life and the lives of those around them.1 Early diagnosis is also important as NP-C is a treatable disease. Treatment can help to manage the symptoms and can slow down the progression of the disease.1,2 Therefore, it is critical that we aim to achieve earlier diagnosis of NP-C.

Who is the Think Again. Think NP-C campaign targeted at?

The Think Again. Think NP-C campaign is targeted at healthcare professionals who might see a patient with NP-C, but are unfamiliar with NP-C and therefore are currently unable to Think NP-C. Once they suspect a patient has NP-C they can then refer that patient on to a specialist centre for diagnostic testing.

The Think Again. Think NP-C campaign is not aimed at those healthcare professionals who already know a lot about NP-C or who carry out the diagnostic tests to confirm a diagnosis of NP-C as they will most likely already be aware of NP-C.

Who can get involved in the Think Again. Think NP-C campaign?

Anyone can get involved to raise awareness of NP-C. This includes, but is not limited to, patient support groups, individual patients and their carers, healthcare professionals and the media.

A number of educational materials have been developed as part of this campaign to support healthcare professionals unfamiliar with NP-C to recognise the key signs and symptoms of the disease.

Who has developed the Think Again. Think NP-C campaign?

The following organisations and individuals have been involved in developing the campaign:

  • International Niemann-Pick Disease Alliance (INPDA)
    The campaign is led by the INPDA, which is an international alliance of non-profit Niemann-Pick Disease patient support organisations. Find out more about the INPDA
  • Advisory committee
    A multidisciplinary advisory committee of leading clinical experts in the field of NP-C provided advice and input into the content of the campaign to ensure it would resonate with healthcare professionals. Find out more about the advisory committee
  • Actelion Pharmaceuticals Ltd
    Financial support to develop the campaign was provided by Actelion Pharmaceuticals Ltd. Actelion Pharmaceuticals Ltd is a biopharmaceutical company which focuses on the discovery, development and marketing of innovative drugs for significant unmet medical needs.

Any questions? Visit our FAQs section to find out more.

References

  1. Patterson M, Hendriksz, Walterfang M, et al. on behalf of the NP-C Guidelines Working Group. Recommendations for the diagnosis and management of Niemann-Pick disease type C: an update. Mol Genet Metab 2012; 106(3):330-44.
  2. Wijburg F, Sedel F, Pineda M, et al. Development of a suspicion index to aid diagnosis of Niemann-Pick disease type C. Neurology 2012;78(20):1560-7.
  3. Vanier M. Niemann-Pick disease type C. Orphanet J Rare Dis 2010;5:16.
  4. Klünemann H, Wraith E, Wijburg F. Niemann-Pick Type C Disease – Report on Results from the Niemann-Pick Type C Patient and Healthcare Professional Survey. Eur Neurol Rev 2011;6(1):12–15.
  5. Mengel E, Klünemann H, Lourenço C, et al. Niemann-Pick disease type C symptomatology: an expert-based clinical description. Orphanet J Rare Dis 2013; 8:166.

 

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