New hope for SMA babies: boosting gene therapy with a Follow-Up drug

NCT ID NCT07444450

Recruiting now Disease control Sponsor: Biogen Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Aug 12, 2026 · Updated 2 times

Summary

This study tests a drug called salanersen in babies with spinal muscular atrophy (SMA) who have already received gene therapy. The drug aims to help the body make more SMN protein, which is needed for muscle function. About 42 babies with two copies of the SMN2 gene will be randomly assigned to get either salanersen or a sham procedure. Researchers will track safety and motor milestones over up to 5.5 years.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: salanersen (BIIB115)
What this could lead to: If it works, this could lead to better motor development and fewer symptoms for babies with SMA who have already had gene therapy.
What could go wrong: This is an early-stage trial with only 42 participants, so results may not apply to all. The sham procedure and lumbar puncture carry risks like discomfort or infection.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for MUSCULAR ATROPHY, SPINAL are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

spinal muscular atrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

MUSCULAR ATROPHY, SPINAL

Contacts and locations

Locations

Neurology Rare Disease Center
RECRUITING

Flower Mound, Texas, 75028, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.