New drug shows promise in preventing SMA in newborns

NCT ID NCT03779334

Ongoing Disease control Sponsor: Hoffmann-La Roche Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jul 10, 2026 · Updated 2 times

Summary

This study tests the drug risdiplam (Evrysdi) in infants up to 6 weeks old who have a genetic diagnosis of spinal muscular atrophy (SMA) but no symptoms yet. The goal is to see if early treatment can help them reach motor milestones like sitting without support. The trial involves 26 participants and is currently active but not recruiting.

What this could mean

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

Active substance: risdiplam (Evrysdi)
What this could lead to: If successful, this could prevent or delay the onset of spinal muscular atrophy in infants diagnosed before symptoms appear, allowing them to achieve normal motor milestones.
What could go wrong: This is a small Phase 2 study with only 26 infants, so results may not apply to all patients. Long-term safety and efficacy are still being evaluated, and some children may still develop symptoms or need ongoing treatment.

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

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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

Chr de La Citadelle

Liège, 4000, Belgium
Hospital das Clinicas - FMUSP_X

São Paulo, São Paulo, 05403-000, Brazil
Kaohsiung Medical University Chung-Ho Hospital

Kaohsiung City, 807, Taiwan
Nemours Children's Hospital

Orlando, Florida, 32837, United States
Russian Children Neuromuscular Center of Veltischev

Moscow, Moscow Oblast, 125412, Russia
Sydney Children's Hospital

Randwick, New South Wales, 2031, Australia
Szpital Gdanskiego Uniwersytetu Medycznego

Gda?sk, 80-952, Poland

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