Condition: Spinal Muscular Atrophy Type I · Sponsor: Gemma Biotherapeutics
GB221 is a gene therapy that delivers a working SMN1 gene to the motor neurons of people with spinal muscular atrophy (SMA) Type 1. This study will evaluate the safety, tolerability and efficacy of GB221 in two groups: 1. participants aged from 2 weeks to younger than 12 months presenting with symptoms of SMA Type 1 who have never received a treatment OR are receiving the drug risdiplam 2. participants aged from 2 weeks to younger than 5 months who are at risk of developing SMA Type 1 (presymptomatic) and have never received treatment OR are receiving the drug risdiplam.
This description comes directly from the study's public registry record.
Denise Reilly · 267-718-7654 · clinical_studies@gemmabiotx.com
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| Hospital de Clínicas de Porto Alegre | Porto Alegre, Rio Grande do Sul, Brazil | Recruiting |
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Source record: clinicaltrials.gov/study/NCT07070999