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

Description

This study will evaluate the safety andtolerability of RGX-111 in patients with Mucopolysaccharidosis Type I with aneurocognitive deficit that are at least 4 months old. This study involvesadministration of the RGX-111, general anesthesia, lumbar puncture, blooddraws, MRI, ultrasound, electrocardiogram, echocardiogram, hearing test, andchart review. If you have questions or would like to learn more about thestudy, please contact the Principal Investigator, Dr. Ficicioglu at (215)590-3376. 

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