Clinical Trial: SPX-01 Gene Therapy in Pediatric Patients With Spinal Muscular Atrophy Type 1 (RISE)

U.S., Oct. 30 -- ClinicalTrials.gov registry received information related to the study (NCT07903376) titled 'SPX-01 Gene Therapy in Pediatric Patients With Spinal Muscular Atrophy Type 1 (RISE)' on Oct. 17.

Brief Summary: The goal of this clinical trial is to learn about the safety, and tolerability of giving an experimental gene therapy called SPX-01 to an infant with spinal muscular atrophy (SMA) type 1. SMA type 1 is a rare inherited disease that causes severe muscle weakness and can affect breathing, swallowing, and movement.

SPX-01 is designed to deliver a working copy of a gene called SMN1, which does not function properly in children with SMA.

The main questions this study aims to answer are:

* What side effects or medical proble...