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A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants With Type1 Spinal Muscular Atrophy

Mise à jour : Il y a 4 ans
Référence : EUCTR2016-000778-40

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)

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