Niemann-Pick Canada

07/19/2025

🇨🇦 Canada – it’s our turn to be heard! 📢

The NPC Survey closes 31 July, and your input is crucial to highlight the needs of Canadian NPC patients and caregivers.

Complete the survey now: Qualtrics Survey | Qualtrics Experience Management

02/28/2025

Did you know there are over 7,000 rare diseases that affect millions of people around the globe? Individually, our numbers are small but together we are loud. Today is Rare Disease Day. Join us in recognizing our fight against ASMD and NPC.

Rare Disease Day is a global movement that unites the voices of 300 million people living with rare diseases. As the INPDA, we are proud to stand together with our member organisations from around the world, working towards a future where no one affected by Niemann-Pick diseases feels alone 💜🩷💚

Our global network is represented in this map, highlighting the strength and unity of our community. Together, we can raise awareness, advocate for better access to care, and drive change for those living with rare diseases.

Join us in making an impact today!

01/09/2025

NPC Study Opportunity: Help us better understand the impact of living with Niemann-Pick type C (NPC) 💡
https://intnpdr.qualtrics.com/jfe/form/SV_8oU4Ytx4DfqM8m2

We know that Niemann-Pick type C (NPC) affects the quality of life for those living with the condition, as well as for their families and caregivers. If you have NPC, or you care for someone who has NPC, we would like to invite you to join our research study and share your experiences.

▪️ What is this Study about?
The purpose of the study is to gain insight into the challenges faced by individuals

09/21/2024

Amazing News for the Niemann Pick community. An FDA approved treatment for Niemann Pick Type C!!

The U.S. Food and Drug Administration approved the first treatment for Niemann-Pick disease type C.

“Today's FDA approval marks a significant milestone for people living with NPC, as it represents the first treatment available for this devastating condition. We are immensely grateful for the unwavering support of the NPC community throughout this journey," said Neil F. McFarlane, President and CEO.

Read the full announcement here: https://bit.ly/3TziV6g

04/23/2024

There is only one way to cure NPC - Together!!
https://notredameday.nd.edu/p2p/365374/dj-love

02/27/2024

10/13/2023

Please join NNPDF, Sanofi and Lurie Children's Hospital for a discussion led by Dr. Josh Baker on Transforming Tomorrow with a Treatment Option for ASMD. See information below.

On behalf of Sanofi and Dr. Baker at Lurie Children’s Hospital, we are pleased to share this invitation with you: Join Sanofi, (in person or virtually), for a discussion led by Dr. Josh Baker on Transforming Tomorrow with a Treatment Option for ASMD on Saturday, October 21st in Oak Brook, Illinois. This discussion is limited to US audience. Learn more and register at t.ly/Sanofi-TransformingTomorrow10.21.23

09/30/2023

October is Niemann-Pick Awareness Month! Share and use our badges on your profiles to spread awareness! Learn more at t.ly/

09/30/2023

October is Niemann-Pick Awareness Month! Share and use our badges on your profiles to spread awareness! Learn more at

08/28/2023

Acid sphingomyelinase deficiency (ASMD) is a lysosomal storage disease caused by deficient activity of acid sphingomyelinase (ASM) enzyme, leading to the accumulation of varying degrees of sphingomyelin.

Here Melissa Wasserstein, MD (Chief, Division of Paediatric Genetic Medicine at Albert Einstein College of Medicine), and Dr Robin Lachmann (Consultant in Metabolic Medicine at UCLH) define in depth the symptoms, cause, and effect of ASMD Niemann-Pick disease type A and type B.

What is ASMD Niemann-Pick disease? Here Melissa Wasserstein, MD (Chief, Division of Paediatric Genetic Medicine at Albert Einstein College of Medicine), and ...