2 results
Approved WMOCompleted
The primary outcome of the study is a 50% reduction in exacerbation rate in patients using TIS once daily (OD). Secondary outcome parameters are lung function (FEV1, FVC), QoL (QOL-B), LTRI-VAS, Leicester cough score), bacterial load in sputum and…
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…