5 results
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…
In a randomized control trial (RCT) with mothers with postpartum depressive symptoms the following main hypotheses will be tested:• Oxytocin will promote more sensitive caregiving during mother-infant interactions and while interacting with a life-…
Primary Objective: To evaluate the efficacy of ION373 in improving or stabilizing gross motor function in patients with Alexander disease Secondary Objectives: To further evaluate the efficacy of ION373 in improving or stabilizing disease…
The aim of this study is to evaluate if a PPT can significantly decrease - or possibly prevent - the collapse of the trachea in newborns with EA and moderate or severe TM. Additionally, the study aims to determine whether the observed effect of PPT…
Primary Endpoint: Feasibility of including patients in the study, randomization and delivery of the intervention. To assess the primary endpoint, we will examine the probability that: (1) a screened patient is eligible; (2) an eligible patient…