New drug could help babies with SMA walk and sit normally

NCT ID NCT07221669

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

First seen Jun 27, 2026 · Last updated Aug 12, 2026 · Updated 5 times

Summary

This study tests a drug called salanersen in babies who have a genetic diagnosis of spinal muscular atrophy (SMA) but no symptoms yet. The drug helps the body make more of a protein that is missing in SMA, which is needed for muscles to work. Researchers want to see if starting treatment early can help babies reach normal milestones like sitting and walking, and prevent or reduce symptoms.

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 successful, this could allow infants with SMA to reach normal motor milestones like sitting and walking, potentially preventing severe disability.
What could go wrong: This is an early-stage, small trial (30 infants) with no placebo group, so results may not be definitive. Long-term safety and efficacy are still unknown.

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

Ann and Robert H Lurie Childrens Hospital of Chicago
RECRUITING

Chicago, Illinois, 60611, United States
Childrens Hospital of Fudan University_Shanghai
RECRUITING

Shanghai, 201122, China
Childrens Hospital of the Kings Daughter Norfolk
RECRUITING

Norfolk, Virginia, 23507, United States
Guangzhou Woman and Children's Medical Center_Guangzhou
RECRUITING

Guangzhou, 5106236, China
Hospital da Crianca de Brasilia
RECRUITING

Distrito Federal, 70684-831, Brazil
Japan Institute for Health Security National Center for Global Health and Medicine
RECRUITING

Tokyo, 162-8655, Japan
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.