4 results
The purpose of the study is to investigate to what extent RO5459072 is safe and well tolerated. It will also be investigated how quickly and to what extent RO5459072 is absorbed, distributed to and removed from the body (this is called…
The purpose of the study is to investigate to what extent RO5459072 is safe and well tolerated.It will also be investigated how quickly and to what extent RO5459072 is absorbed, distributed to and removed from the body (this is called…
Primary ObjectiveThe primary objective is to assess safety and tolerability, describe the dose limiting toxicities (DLTs), and determine the maximum tolerated dose (MTD) or the maximum administered dose (MAD; in the absence of exceeding the MTD) for…
The primary objective of the study is the following:• To characterize the safety and tolerability of DNL310 in pediatric subjects with MPS II (all cohorts)The secondary objectives of the study are as follows:• To characterize the CNS effects of…