Offered by: Nemours Children's
Location: Orlando, Fla.
Phase I, Open-Label, Dose Comparison Study of AVXS-101 for Sitting but Non-ambulatory Patients with Spinal Muscular Atrophy.
The purpose of this study is to find out if this one-time therapy can replace the SMN protein with one that is fully functional.
Children under five years of age with SMA Type 1.
48–72 hour stay in Nemours Children’s Hospital, Florida for the spinal injection and 15 follow-up visits over 12 months. Procedures and tests to be done include a single injection by lumbar puncture, EKG, physical exam, blood and urine samples and various muscle tests.
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