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

Description

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. If you have questions or would like to learn more about the
study, please contact the Principal Investigator, Dr. Ficicioglu at (215)
590-3376. 

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