We Care March 2024

We Care March 2024

Spring Is Here! Let’s Spread Seeds of Hope! Join us this March for our monthly We Care virtual gathering celebrating the incredible strength of our MPS community. We’ll kick things off by sharing humble brags about our brave MPS warriors and their triumphs, no...
Denali MPS IIIA Clinical Study

Denali MPS IIIA Clinical Study

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...
Clinical Trial RGX-111 for MPS I Halted

Clinical Trial RGX-111 for MPS I Halted

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...
May is the month for MPS awareness!

May is the month for MPS awareness!

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...
National Strategy for Drugs for Rare Diseases

National Strategy for Drugs for Rare Diseases

$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...