Offered by: Nemours Children's
Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion.
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 six months of age with SMA Type 1.
48–72 hour stay in Nemours Children’s Hospital, Florida for the injection and 15 follow-up visits over 12 months. Procedures and tests to be done include a single injection by IV through arm or leg, ECG, echo, physical exam, blood and urine samples and various muscle tests.
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