For more information, ask your healthcare provider or pharmacist
The Bottom Line GLP-1 medications can be highly effective tools for managing obesity and diabetes, but cost remains a significant barrier for many people
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Patients Treated With REGENXBIO's MPS II Gene Therapy RGX-121 Sustain 82% Median Reduction in CSF HS D2S6 Levels Through 1 Year September 10, 2025 According to a new data update from REGENXBIO's pivotal phase 1/2/3 CAMPSIITE clinical trial (NCT03566043) evaluating clemidsogene lanparvovec (RGX-121), an investigational adeno-associated virus (AAV) vector-based gene therapy for mucopolysaccharidosis type 2 (MPSII, also known as Hunter syndrome), 13 patients treated in the pivotal phase have shown an 82% reduction in heparan sulfate (HS) D2S6 levels in the cerebrospinal fluid (CSF) through 1 year posttreatment
For 503B facilities specifically, the FDA maintains a list of registered outsourcing facilities at FDA.gov
154 However, further research is needed to evaluate their safety and efficacy in clinical settings
