4 results
The overall objective is to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in male and femaleparticipants less than or equal to 6 months of age with IOPD.
Part 1: To evaluate the efficacy and safety of imetelstat in transfusion dependent subjects with low or intermediate-1 risk MDS that is relapsed/refractory to ESA treatment.Part 2: To compare the efficacy, in terms of red blood cell (RBC)…
Long-term safety and pharmacokinetics (PK) of neoGAA
1. Assessing the efficacy of IVIg in autoimmune epilepsy, both clinically and serologically.2. Identifying an objective marker of therapy response in epilepsy, measuring cortical excitability by TMS-EEG/EMG, in vivo.3. Providing evidence that…