Type 2 spinal muscular atrophy

Clinical trials for Type 2 spinal muscular atrophy explained in plain language.

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Gene therapy hope for SMA kids: early trial launches

Disease control Ongoing

This early-stage trial tests a new gene therapy called NKG001 in 21 children under 5 with spinal muscular atrophy (SMA). The therapy is given as a single dose, either through a vein or combined with a spinal injection. The main goal is to check safety and find the best dose, not …

Matched conditions: TYPE 2 SPINAL MUSCULAR ATROPHY

Sponsor: Nikegen Pharmaceutical (Hangzhou) Company Limited • Aim: Disease control

Last updated Jun 27, 2026 13:00 UTC

Type 2 spinal muscular atrophy

Get alerted when new Type 2 spinal muscular atrophy trials appear

Gene therapy hope for SMA kids: early trial launches