Gene therapy hope for babies with rare muscle disease
NCT ID NCT07070999
First seen Jun 27, 2026 · Last updated Jun 30, 2026 · Updated 2 times
Summary
This study tests a single dose of GB221, a gene therapy that delivers a working SMN1 gene, in infants aged 2 weeks to under 12 months with spinal muscular atrophy (SMA) Type 1. The goal is to see if it is safe and helps improve motor function. The trial includes both symptomatic and presymptomatic babies, and will monitor side effects and developmental milestones.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
GB221 (a gene therapy that delivers a working SMN1 gene)
What this could lead to
If it works, this could point toward a treatment that helps infants with SMA Type 1 gain motor skills and avoid permanent ventilation.
What could go wrong
This is an early phase 1-2 trial with only 22 participants, so results may not apply to everyone. There are risks of side effects like immune reactions or lab abnormalities.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Hospital de Clínicas de Porto Alegre
RECRUITINGPorto Alegre, Rio Grande do Sul, 90035-903, Brazil