New gene therapy trial offers hope for babies with rare muscle disease
NCT ID NCT07070999
First seen Dec 26, 2025 · Last updated May 23, 2026 · Updated 19 times
Summary
This study tests a single dose of a gene therapy called GB221 in babies aged 2 weeks to under 12 months who have spinal muscular atrophy (SMA) type 1, a severe muscle-weakening disease. The therapy delivers a working gene to help nerve cells control muscles. Researchers will check if it is safe and helps babies breathe and move better. The trial includes both babies who already have symptoms and those at risk but not yet showing signs.
Disclaimer
Read more
Show less
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for SPINAL MUSCULAR ATROPHY TYPE I are added.
Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
Genom att skicka in godkänner du våra Användarvillkor
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
Hospital de Clínicas de Porto Alegre
RECRUITINGPorto Alegre, Rio Grande do Sul, 90035-903, Brazil
Conditions
Explore the condition pages connected to this study.