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...
Every year May 15 is observed internationally, to raise awareness against mucopolysaccharidoses (MPS). MPS are rare genetic disorders in children and adults caused by the absence of a specific enzyme. There are 7 types of MPS. It is estimated that 1 in every 25,000...
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...
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...
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...
