Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1

Recruiting

Phase 1 — Early testing in a small group of people (usually 20–80) to evaluate safety, determine safe dosage, and identify side effects.

Trial locations(1 site)

Brazil

Hospital de Clínicas de Porto Alegre, Porto Alegre, Rio Grande do Sul