Mucopolysaccharidosis type 2 (MPS2), severe form (MPS2S), is associated with a massive accumulation of glycosaminoglycans and a wide variety of symptoms including a rapidly progressive cognitive decli...
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Connect with organizations supporting the mucopolysaccharidosis type 2, severe form community
Helpful links for rare disease information and support
Questions that may be helpful when speaking with your healthcare team
Connect with organizations supporting the mucopolysaccharidosis type 2, severe form community
Helpful links for rare disease information and support
Questions that may be helpful when speaking with your healthcare team
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Research studies investigating treatments and therapies for this condition.
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Data from ClinicalTrials.gov Feb 8, 2026
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AI-curated news mentioning mucopolysaccharidosis type 2, severe form
Updated Feb 13, 2026
FDA issues a complete response letter for REGENXBIO's gene therapy RGX-121 intended for mucopolysaccharidosis type 2 (MPS II), also known as Hunter syndrome. The agency raised concerns regarding clinical trial eligibility criteria and differentiation between disease types.
The FDA issued a complete response letter to REGENXBIO for its gene therapy RGX-121, intended for mucopolysaccharidosis type 2 (MPS II), also known as Hunter syndrome. The agency raised concerns regarding clinical trial eligibility criteria and its ability to differentiate between disease types.
Regenxbio's RGX-202 gene therapy for Duchenne muscular dystrophy (DMD) demonstrated significant improvements in patient outcomes, exceeding expected disease progression at 12 and 18 months post-treatment in a pivotal trial. The company plans to submit a Biologics License Application (BLA) in mid-2026 via an accelerated approval pathway.