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Research Studies

Metabolic Disease Program Research Studies

Find research studies available to children cared for by the Metabolic Disease Program team.

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Recruitment Status

Eligible age

Clinical trial phase

RGX-111 Gene Therapy in Patients With Mucopolysaccharidosis Type I (MPS I)

This study will evaluate the safety and tolerability of RGX-111 in patients with Mucopolysaccharidosis Type I with a neurocognitive deficit that are at least 4 months old. This study involves administration of the RGX-111, general anesthesia, lumbar puncture, blood draws, MRI, ultrasound, electrocardiogram, echocardiogram, hearing test, and chart review.

Phase: Phase I, Phase II

Actively recruiting: No

Category: Adults, Children

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