by adminmps | Nov 25, 2024 | News, Resources
SickKids, Toronto has expanded their innovative mental health support program, specifically designed with MPS families in mind. They understand the unique challenges and strengths of families impacted by MPS, and are committed to providing compassionate, tailored...
by adminmps | Jan 22, 2024 | News, Resources
Inform Rare has launched a new resource – CommuniKIDS – a template to help researchers convey clinical trial results back to participating youth and their families in plain easy to understand language. CommuniKIDS, funded in 2021 by CHILD-BRIGHT, takes its...
by adminmps | Dec 13, 2023 | Clinical Trials, News, Research
December 12, 2023 Dear Sanfilippo syndrome Type A Community, We are pleased to share that a Phase 1/2 clinical study of DNL126, an investigational enzyme replacement therapy designed to address the behavioral, cognitive, and physical symptoms in Sanfilippo syndrome...
by adminmps | Nov 9, 2023 | Clinical Trials, News
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...
by adminmps | Apr 5, 2023 | News
$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...
by adminmps | Apr 4, 2023 | Clinical Trials, News
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....
