News Updates

Keep up-to-date with the latest news, updates, and initiatives from the International Niemann-Pick Disease Alliance (INPDA), working together to support families and drive progress worldwide.

Newsletter

We are proud to present the INPDA International Niemann-Pick Disease Alliance Newsletter, which serves as a way to present exciting and informative updates from each one of our member groups, in a dynamic format.

INPDA President, Sandy Cowie writes: “…this newsletter will serve as a communication tool for all of the members of the INPDA, to share information and updates. It will be released in an electronic format 3 times per year which allows for each edition to be full of news which will likely be of interest to the global Niemann-Pick community. We hope you enjoy it…”

To subscribe to the INPDA Newsletter, and ensure you never miss an update, please contact Sarah Jannetta at sarah. jannetta@inpda.org.

You can view previous editions at the links below:

Publications

A PATIENT SURVEY REPORT

NP-C_REPORT METABOLICS

NP-C_REPORT_NEUROLOGISTS

NP-C_REPORT_PAEDIATRICIANS

NP-C_REPORT_PSYCHIATRISTS

MYELIN DEFECTS IN NIEMANN–PICK TYPE C DISEASE: MECHANISMS AND POSSIBLE THERAPEUTIC PERSPECTIVES

INTRATHECAL 2 HYDROXYPROPYL-Β CYCLODEXTRIN DECREASES NEUROLOGICAL DISEASE PROGRESSION IN NIEMANN-PICK DISEASE,TYPE C1: A NON RANDOMISED, OPENLABEL, PHASE 1–2 TRIAL

THINK AGAIN. THINK NP-C EDUCATIONAL
SLIDE DECK

Pfrieger’s Digest Archives

Clinical Care and Guidelines

CONSENSUS CLINICAL MANAGEMENT GUIDELINES FOR NIEMANN-PICK DISEASE TYPE C (2018)

RECENT ADVANCES IN THE DIA GNOSIS AND TREATMENT OF NIEMANN-PICK DISEASE TYPE C IN CHILDREN: A GUIDE TO EARLY DIAGNOSIS FOR THE GENERAL PEDIATRICIAN (2015)

RECOMMENDATIONS FOR THE DIA GNOSIS AND MANAGEMENT OF NIEMANN-PICK DISEASE TYPE C (2012)

ELSEVIER: RECOMMENDATIONS ON THE DIA GNOSIS AND MANAGEMENT OF NIEMANN-PICK DISEASE (2009)

ARGENTINEAN CONSENSUS ON THE DIA GNOSIS AND TREATMENT OF NIEMANN-PICK DISEASE TYPE C (2023)

Find a Diagnostic Centre

Looking for a diagnostic centre in your country?

Orphanet is a unique resource, aiming to provide high-quality information on rare diseases, and ensure equal access to knowledge for all stakeholdes.

Think Again. Think NPC Campaign

Think Again. Think NPC is a groundbreaking awareness campaign led by the International Niemann-Pick Disease Alliance (INPDA), an umbrella group of non-profit Niemann-Pick disease patient support organisations from across the world.

The campaign aims to reduce the time to diagnosis of NPC, with particular regard to healthcare professionals who may be unfamiliar with the condition and therefore may struggle to recognise the key signs and symptoms of the disease. By increasing awareness, and encouraging those people to Think Again. Think NPC we will help patients by speeding up diagnosis and subsequent access to treatment and support.

Campaign Resources:

ATAXIA

HEPATOSPLENOMEGALY

DYSARTHRIA

ORGANIC PSYCHOSIS

GELASTIC CATAPLEXY

VERTICAL SUPRANUCLEAR GAZE PALSY