5 results
The primary objectives of this study are: • To determine the longterm safety and tolerability of rhHNS administered via an intrathecal (IT) route once monthly for 8 years in patients with MPS IIIA, who have received and tolerated 6 months of…
The study will be performed in 2 parts, Part 1 and Part 2). The purpose of the study is to investigate how quickly and to what extent Olesoxime is absorbed and eliminated from the body. In addition, it will be investigated how safe Olesoxime is and…
Main objective: The main objective of this open-label, single arm study is to further characterize the safety, tolerability and effectiveness profile of olesoxime in SMAThe primary safety objective for this study is as follows:To evaluate the safety…
Objectives: For patients with connective tissue disease-associated pulmonary arterial hypertension (CTD-PAH) enrolled in this study, the objectives are as follows:Primary: -To assess the efficacy of bardoxolone methyl relative to placebo.Secondary…
To provide continuing open-label treatment with bardoxolone methyl as part of this extended access program while collecting ongoing safety and tolerability data of bardoxolone methyl.