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A Study to investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics And Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients

Mise à jour : Il y a 4 ans
Référence : EUCTR2016-000750-35

Femme et Homme

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Extrait

Part 1: •To evaluate the safety, tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of RO7034067 in patients with Type 2 and Type 3 (ambulant or non-ambulant) SMA, and to select the dose for Part 2 of the study Part 2: •To evaluate efficacy of RO7034067 compared to placebo in terms of motor function in Type 2 and non-ambulant Type 3 SMA patients, as assessed by the change from baseline in the total score of the Motor Function Measure (MFM) at 12 months

Critère d'inclusion

  • Spinal Muscular Atrophy (SMA)

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