News Feed

News from the MPS Community, industry and research.
To view posts by category, click on your chosen category which appears after the date in any of the posts.

The Rare Sibling Experience: Sibshops Support

Sep 6, 2022 | News, Resources

Listen to a talk about the sibling perspective and the support available with Emily Holl, the Director of the Sibling Support Project. Emily is a...

Results Of the Consultation On the PMPRB Price Review

Sep 6, 2022 | News

On June 30, 2022, The Patented Medicine Prices Review Board (PMPRB) issued a Notice and Comment on its proposed approach for...

Anaesthesia Recommendations For Rare Diseases

Sep 6, 2022 | News, Resources

Want to find out more about anaesthesia and how it relates to rare diseases? OrphanAnesthesia publishes anaesthesia recommendations for rare...

MPS II is added to New Born Screening in the USA

Sep 6, 2022 | News

The Department of Health and Human Services (DHHS) in the United States has approved adding MPS II as a condition to the recommended uniform...

Ultragenyx Takes Over Gene Therapy For Sanfilippo Syndrome Type A (MPS IIIA)

Sep 6, 2022 | Clinical Trials, News, Research

In our March edition we reported that Abeona Therapeutics had decided to end enrollment for their clinical trial for MPS IIIA children. We are now...

Another Potential Gene Therapy to Combat Against Hunter Syndrome MPS II

Sep 6, 2022 | Clinical Trials, News, Research

We are excited to share the news that REGENXBIO Inc. has announced its intention to file a Biologics License Application (BLA) in 2024 using the...

Gene Therapy For Inherited Metabolic Diseases Explained

Sep 6, 2022 | News, Resources

Ever wondered… How does gene therapy work?Why does it work better for some conditions that others?What is the difference between in vivo and ex vivo...