2 results
Approved WMOPending
The primary objectives of this study are to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA), as measured by change in Children's Hospital of Philadelphia…
Approved WMOPending
Main objective:• Evaluate the efficacy of IPN60130 monotherapy compared with placebo recipients in inhibiting new HO volume in adult and paediatric participants with FOP as assessed by low-dose WBCT (excluding the head)• Evaluate the safety of…