International Niemann–Pick Disease Alliance

International Niemann-Pick Disease Registry

The International Niemann-Pick Disease Registry (INPDR) is a global disease specific patient registry – a ground-breaking initiative that can harness patient power for the benefit of the Niemann-Pick disease community, as well as potentially other rare diseases.

Aside from the research, tests, results, diagnosis odyssey, lack of awareness and so on, living with a rare disease can be an emotionally, mentally and financially draining experience, not to mention isolating.

Understandably many families are just trying to deal with a life-changing diagnosis, focus on the positives and enjoy their time together

Perhaps the idea of databases, longitudinal studies and research that may not have an immediate impact is low on the list of priorities but we’re asking patients and their families to reconsider.

We welcome all patients diagnosed with any type of NPD worldwide. Patients can participate in the registry regardless of whether or not they are involved in other clinical studies and trials.

If you have any questions at all, please do contact us.

What is the INPDR?

There are two parts to the registry; clinician reported database and patient reported database.

The clinician reported data captures demographic, diagnosis, medical history and treatment details, while the patient reported data looks at the real-world experience of living with the disease: quality of life, health economics and disease impact assessment. Both types of data are held securely and anonymously.

The data held in the registry will:

  • contribute to research,
  • show the natural history of all types of Niemann-Pick disease,
  • establish genotype-phenotype correlations,
  • support recruitment to interventional studies,
  • educate health professional,
  • improve standards of diagnosis, clinical management and care,
  • empower patients.

The Clinician Reported Database and Patient Reported Database have just been redeveloped and launched to give a much better experience for those adding their data but also to make sure the quality of data is of the highest standard and useful for vital research.

A global gateway

Before the INPDR was developed patients with rare diseases were often told that they may never meet anyone with the same condition – now there are some amazing advocates and networks that have helped patients and their families to connect across the World.

Patient registries were often unique to a certain treatment study and exclusive to the pharmaceutical company – the INPDR breaks that mould by bringing together vital information from patients across the World and providing equitable access to the data.

With over 300 ASMD and NPC patient records from 13 different countries, the INPDR is attracting interest from clinicians, researchers and scientists hoping to learn and improve understanding, diagnosis and therapy for NPD patients and their families.

Several more clinicians across the globe are going through the process of consenting patients so they can add to the data.

Researchers and industry are exploring more ways they can use the data to learn and progress – we have a stringent Data Access Request process so we can make sure the INPDR and those who use the data within directly contribute to progress for the Niemann-Pick Disease community.

A lasting legacy

Signing up to the INPDR is an opportunity for patients and families to contribute to a legacy.

By sharing their experience of the disease, it will help to make a difference for families, the NPD community and future generations – maybe one day helping to find a cure

Patient records are increasing but the INPDR needs more people to sign up in order to harness the power of patient data.

Your information can be held in either the CRD, the PRD, or in both sections on the INPDR.

We welcome all patients diagnosed with any type of NPD worldwide. Patients can participate in the Registry regardless of whether or not they are involved in other clinical studies and trials.

We urge patients and their families living with Niemann-Pick Disease to speak to their clinicians and let them know they would like their data added to the Registry.

 

Join the Registry

Calling all patients and carers! We've created a global registry of all NPD patients - if you're not enrolled yet, join us now...

Think Again. Think NP-C

Join our international campaign to improve diagnosis of NP-C!