2 results
Approved WMORecruiting
We aim to prevent or reverse developmental regression in patients with Kleefstra syndrome. We hypothesise that the regression we observed is the result of a psychotic disorder, which isnot properly recognised by clinicians and often not treated…
Approved WMOPending
The purpose of this study is to evaluate the safety and efficacy of ALXN1720 for the treatment of gMG in adults with autoantibodies against AChR (AChR+). The study will also evaluate the safety and performance of the administration device (the PFS-…