Femme et Homme
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Extrait
Part 1 •To evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of RO7034067 in infants with Type 1 SMA and to select the dose for Part 2 Part 2 •To assess the efficacy of RO7034067 measured as the proportion of infants sitting without support after 12 months of treatment
Critère d'inclusion
- Type 1 Spinal Muscular Atrophy (SMA)