Support for families. Research for a cure.
Our support and education programs assist those affected in managing the diagnosis and progression of these rare, often devastating, disorders. Funds raised through donations and special events help fund research that provides hope for a brighter future for all children born with lysosomal diseases.
Current News and Events
REGENXBIO Announces Positive Interim Data and Update for PHASE I/II Trial of RGX-121 for the Treatment of MPS II
REGENXBIO announced interim data from Cohorts 1 and 2 of the ongoing Phase I/II trial of RGX-121 for the treatment of patients up to 5 years old diagnosed with Mucopolysaccharidosis Type II (MPS II). In [...]