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Protocol Number: AVXS101CL302Official Title: Phase 3, OpenTable, Single, Singles Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering

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What is phase 3 open-label single-arm?

Phase 3 open-label single-arm is a type of clinical trial where all participants receive the same treatment and there is no placebo or control group.

Who is required to file phase 3 open-label single-arm?

Researchers conducting phase 3 open-label single-arm clinical trials are required to file the results.

How to fill out phase 3 open-label single-arm?

Researchers must accurately report the findings and outcomes of the phase 3 open-label single-arm clinical trial.

What is the purpose of phase 3 open-label single-arm?

The purpose of phase 3 open-label single-arm is to evaluate the efficacy and safety of a treatment in a larger group of participants.

What information must be reported on phase 3 open-label single-arm?

Information such as study design, participant demographics, treatment details, and study results must be reported on phase 3 open-label single-arm.

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