2 results
Approved WMOCompleted
The primary objective is to evaluate the effects of administration of neflamapimod (VX-745) for 24-weeks on immediate and delayed recall aspects of episodic memory, as assessed by the Hopkins Verbal Learning Test * Revised (HVLT-R) in subjects with…
Approved WMORecruiting
The primary aim of the trial is to demonstrate the non-inferiority of CSL222 (formerly AMT-061) (2 × 1013 gc/kg) during the 52 weeks following establishment of stable factor IX expression (months 6 to 18) post-treatment (CSL222 (formerly AMT-061))…