INPDR International Niemann-Pick Disease Registry

21/08/2026

We have extended the survey deadline, giving one last chance to be part of "Shaping the Future of the Patient Reported Database" - please click the link below to add your voice to what we are looking to do here at the INPDR International Niemann-Pick Disease Registry:

https://intnpdr.qualtrics.com/jfe/form/SV_8CzNWzBm8v0jwYm?fbclid=IwY2xjawT1RXhwZG9mAWV4dG4DYWVtAjEwAGJyaWQRMUVxNWJoa1JMWnpzV29vMmpzcnRjBmFwcF9pZBAyMjIwMzkxNzg4MjAwODkyAAEe0-pPRkOYDU9_K4XtDgpEj7TBdB8icKM_cuJLY4EWVlaFKmucU4BSYUCak04_aem_29rik3XkWlYVqqkBUU4kUA

19/08/2026

In this video, Solomon Mbua, Global Lead of the INPDR, shares more about the Patient Reported Database (PRD), the valuable discussions held during PRD Focus Group sessions at the Argentina conference and the Asia-Pacific Meeting for Niemann-Pick Related Diseases (APMRF), and why we're conducting the PRD User Experience Survey.

At both events, community members told us they would like more opportunities to share their views and help shape the future of the PRD.

We know not everyone can attend conferences, which is why this survey is so important. It gives people across the global Niemann-Pick diseases community the opportunity to share their perspectives, experiences, and ideas, wherever they are.

Whether you're already participating in the PRD, have considered joining, or are hearing about it for the first time, we'd like to hear from you.

Your feedback will help us better understand the community's needs and identify opportunities to improve the PRD for everyone.

🔗 Survey link: https://intnpdr.qualtrics.com/jfe/form/SV_8CzNWzBm8v0jwYm
Survey closes on the 21st of August 2026.

14/08/2026

As part of our commitment to continually improving the Patient Reported Database (PRD), we're inviting feedback from across the Niemann-Pick diseases community.

Whether you're an existing participant, have thought about joining, or are learning about the PRD for the first time, your perspective matters.

Your feedback will help us better understand community awareness, experiences, and expectations of the PRD, and identify ways to make it more useful and accessible in the future.

If you haven't already completed the survey, we'd love to hear from you.

🔗 Survey link: https://intnpdr.qualtrics.com/jfe/form/SV_8CzNWzBm8v0jwYm
Survey closes on the 21st of August 2026.

11/08/2026

Your experience can help improve the PRD.

The INPDR PRD User Experience Survey is now open, and we'd love to hear from you.

The PRD exists to capture the experiences and perspectives of people affected by Niemann-Pick diseases, so it's important that we regularly ask the community how we can make it better.

You don't need to be enrolled in the PRD to take part.

Whether you're a participant, a caregiver, a family member, or simply someone interested in learning more, your feedback will help us understand:
• what works well
• what could be improved
• what barriers may exist to participation
• how we can better support the community

Help us ensure the PRD reflects the needs of the people it was created to serve.

🔗 Survey link: https://intnpdr.qualtrics.com/jfe/form/SV_8CzNWzBm8v0jwYm

The most powerful, simple and trusted way to gather experience data. Start your journey to experience management and try a free account today.

07/08/2026

We're pleased to launch the INPDR PRD User Experience Survey, running from the 7th to 21st of August.

In the video below, Shaun Bolton explains what the Patient Reported Database (PRD) is, why it matters, and why we're asking for your feedback.

Whether you:
✅ currently take part in the PRD
✅ have participated in the past
✅ know about the PRD but haven't joined
✅ have never heard of it before..we want to hear from you.

The survey is designed to help us better understand the community's experience, awareness, perceptions, and expectations of the PRD. Your feedback will help guide future improvements and ensure the PRD continues to meet the needs of people affected by Niemann-Pick diseases.
Every perspective matters.

📅 Survey open: 07-21 August
🔗 Take the survey here: https://intnpdr.qualtrics.com/jfe/form/SV_8CzNWzBm8v0jwYm

03/08/2026

At the recent APMRF Conference, participants in the PRD Focus Group sessions shared an important message:
💬 They would like to understand how information collected from the PRD helps support further research and development.

We hear you.

The Patient Reported Database (PRD) is designed to capture the experiences, priorities, and challenges of patients and caregivers.

These insights help researchers better understand disease burden, quality of life, treatment experiences, and unmet needs.
But collecting data is only part of the journey.

The real value comes from transforming that information into research, evidence, and insights that can support:
🔹 Better understanding of Niemann-Pick disease
🔹 Patient-centred research priorities
🔹 Therapeutic development
🔹 Improved care and support for families

We are committed to helping our community better understand how registry data contributes to research and how patient and caregiver voices help drive progress.

Want to explore some of the outputs and publications generated through INPDR data and collaborations?
📚 Resources & Publications: https://inpdr.org/registry-services/resources/

31/07/2026

Every experience shared through the INPDR Patient Reported Database (PRD) helps build a more complete picture of life with Niemann-Pick disease.
Patient-reported outcomes are especially important in rare diseases, where traditional clinical measures and biomarkers may not fully capture the realities of living with a condition.

By collecting information directly from patients and caregivers, the PRD helps to:
🔹 Validate and complement clinical findings
🔹 Identify the symptoms and challenges that matter most to families
🔹 Demonstrate treatment effectiveness from the patient perspective
🔹 Highlight unmet needs that may otherwise go unrecognised
🔹 Support the development of future therapies and care approaches

As interest in patient-focused research continues to grow, industry partners, researchers, and healthcare professionals are increasingly looking to patient-reported experiences and outcomes to better understand the real-world impact of disease and treatment.

By sharing their experiences, patients and caregivers are helping to shape the future of research, care, and therapeutic development for the Niemann-Pick community.

🧡 Every voice matters. Every response makes a difference.

30/07/2026

We're pleased to share that INPDR has been accepted onto RareMap, helping to increase the visibility of the Registry and making it easier for people across the rare disease community to discover and access this important global resource.

RareMap, developed in partnership with LifeArc and Rare Disease Research UK (RDR UK), connects individuals, families, healthcare professionals, researchers, and organisations with trusted rare disease information, services, and support networks.

As a global registry for Niemann-Pick diseases, INPDR brings together patient and clinician-reported data to deepen understanding of the disease, support research, and help accelerate progress towards improved care and future treatments. Being included on RareMap will help raise awareness of the Registry and the vital role patient data plays in advancing rare disease research.

We're proud to join the growing RareMap community and look forward to connecting with even more individuals and organisations worldwide.

🔗 Explore RareMap and learn more about INPDR: https://raremap.co.uk/organisation/international-niemann-pick-disease-registry/

We are an independent organisation dedicated to improving understanding and outcomes in Niemann-Pick diseases. As part of this work, we develop and manage disease-specific registries—structured platforms that systematically collect and curate patient and clinical data.​ ​Through these registri...

30/07/2026

📊 The INPDR Patient Reported Database (PRD) continues to grow, helping us build a deeper understanding of the lived experience of Niemann-Pick disease.

Today, the PRD includes:
✅ Approximately 130 NPC participants enrolled
✅ More than 600 completed survey responses
✅ 70 child caregivers sharing their experiences
✅ 60 adult caregivers contributing vital insights
✅ 10 self-reporting individuals living with NPC

Through participant surveys, the PRD collects valuable information about:
🧡 Quality of life
🧡 Symptoms and disease burden
🧡 The unmet needs of patients and families

Every survey completed helps create a richer picture of life with NPC and ASMD, ensuring that patient and caregiver experiences can help inform future research, clinical understanding, and advocacy efforts.

Thank you to everyone who has taken part and helped make their voice count.

30/07/2026

Update from Zevra regarding Arimoclomol for the treatment of Niemann-Pick Disease Type C in the European Union

This week, Niemann-Pick UK and The INPDA International Niemann-Pick Disease Alliance met with Zevra Therapeutics following the disappointing CHMP opinion on the EU marketing authorisation for arimoclomol:

https://www.npuk.org/2026/07/30/update-from-zevra-regarding-arimoclomol-for-the-treatment-of-niemann-pick-disease-type-c-in-the-european-union/

Zevra reaffirmed its commitment to the NPC community and its intention to request a re-examination.

Further updates will be shared when available, however if you have any questions please contact the NPUK Team by phone on 0191 415 06 93 or by email at [email protected].