3 results
The primary aim of the study is to compare the effect of PRO symptom monitoring with standard care on QoL both during and up to 1 year after treatment. Secondary aims are studying the effect of PRO symptom monitoring compared to standard care on the…
The objective of the study is to assess the safety and tolerability, and to characterize the pharmacokinetics (PK) and pharmacodynamics (e.g., Cerebrospinal fluid [CSF] levels of IL-1β, TSPO positron emission tomography [PET] imaging) of selnoflast…
Objectives for both the Treatment Period and Extension Period:Primary Objectives: - To assess safety and tolerability of the study medicine in patients with later onset (e.g., Type 2 and Type 3) spinal muscular atrophy (SMA)- To assess the efficacy…