Clinical Trial RGX-111 for MPS I Halted
We are disappointed to share that REGENXBIO will no longer be moving forward with the development of RGX-111 for the treatment of MPS I.
National Strategy for Drugs for Rare Diseases
Health Minister Jean-Yves Duclos announced measures for the first-ever National Strategy for Drugs for Rare Diseases, investing up to $1.5 billion over three years.
Post-Trial Access TAK-609
Takeda has announced a Post-Trial Access program for patients currently enrolled in extension trials for TAK-609, a treatment for MPS II Hunter syndrome.
EVERYBODY GRIEVES: Webinar Recording
Grief is considered a "taboo" topic and should be avoided in conversation. As a result, even fewer people know how to respond to and process grief –...
Run for Raza
On September 17, 2022, a group of my family and close friends were kind enough to come together to run the Tough Mudder Obstacle Race in memory of...
Why is it imperative to recognise MPS early?
Kim Angel, Executive Director, Canadian MPS Society explains why recognising MPS early can save a child’s life.
The Rare Sibling Experience: Sibshops Support
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
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
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
The Department of Health and Human Services (DHHS) in the United States has approved adding MPS II as a condition to the recommended uniform...