November 8, 2023 Dear MPS I community, This afternoon REGENXBIO Press Release 8Nov2023, we announced a corporate restructuring that impacts our development of potential AAV gene therapies for MPS I. We are disappointed to share that REGENXBIO will no longer be moving...
$1.5 Billion Canadian National Strategy for Drugs for Rare Diseases The Health Minister Jean-Yves Duclos announced measures in support of the first-ever National Strategy for Drugs for Rare Diseases, with an investment of up to $1.5 billion over three years. This will...
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. This program will provide continued access for patients who have benefited from treatment on existing studies....
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 – both their own and that of others. Grief can be experienced not only after someone’s passing but also can...
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 Raza Amin and the MPS Society. Tough Mudder is a team building endurance event series in which participants attempt...
CTV Kitchener. has featured a story on one of our children, Joseph Saliba, who lives with Sanfilippo Syndrome to raise awareness of MPS. There is no cure for this type of MPS but there is a trial underway for gene therapy, that could help prolong life. The trial,...
