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
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Support groups and aftercare programs provide ongoing support and help prevent relapse
However, with the markets enthusiasm for low-toxicity polypeptide extracts derived from traditional Chinese medicine, polypeptide tyrosinase inhibitors may be the main direction in the future
But these were limited by: Short duration (26 weeks) Advanced disease state Using a prodrug variant, similar to but slightly distinct from Dihexa And even then, there were4/5) Then theres what people report experiences in the real world with personal experimentation
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