SPINAL MUSCULAR ATROPHY TYPE I

Clinical trials for SPINAL MUSCULAR ATROPHY TYPE I explained in plain language.

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Trials to join now! 1 Not yet recruiting 1 Completed 1

One-Time gene infusion aims to save babies from devastating muscle disease

⭐️ CURE ⭐️ Completed

This study tested a single-dose gene replacement therapy for babies under 6 months old with a severe form of spinal muscular atrophy (SMA Type 1). The main goal was to see if the treatment could help these infants achieve the milestone of sitting up on their own for at least 10 s…

Matched conditions: SPINAL MUSCULAR ATROPHY TYPE I

Phase: PHASE3 • Sponsor: Novartis Gene Therapies • Aim: ⭐️ CURE ⭐️

Last updated Mar 30, 2026 14:29 UTC

SPINAL MUSCULAR ATROPHY TYPE I

Get alerted when new SPINAL MUSCULAR ATROPHY TYPE I trials appear

One-Time gene infusion aims to save babies from devastating muscle disease