4 results
The study objectives are to determine the incidence rate of FVIII inhibitors, frequency of adverse events (AEs), and serious adverse events (SAEs) associated with the use of CSL627, to evaluate the PK of 50 IU/kg CSL627, and to evaluate the efficacy…
To evaluate the efficacy of rVIII-SingleChain in the treatment of major andminor bleeding events based on the investigator*s 4-point assessment scale
The objective of the study is to assess the long-term safety and efficacy of intravenous ATB200 co-administration with oral AT2221 in adult subjects with late-onset Pompe disease.
The primary objective is to test the hypothesis that rhPTH(1-84) treatment can result in superior improvements in the symptoms of hypoparathyroidism as assessed by the Hypoparathyroidism Symptom Diary (HPT-SD) symptom subscale compared with standard…