Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Biogen
This study was conducted and the protocol was registered by Ionis Pharmaceuticals, Inc..
In August 2016, sponsorship of the trial was transferred to Biogen.
Age
0–0
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
Be born (gestational age) between 37 and 42 weeks
Be medically diagnosed with spinal muscular atrophy (SMA)
Have Survival Motor Neuron2 (SMN2) Copy number = 2
Body weight equal to or greater than 3rd percentile for age using appropriate country-specific guidelines
Be able to follow all study procedures
Reside within approximately 9 hours ground-travel distance from a participating study center, for the duration of the study
You may not be if
Hypoxemia (oxygen \[O2\] saturation awake less than 96% or O2 saturation asleep less than 96%, without ventilation support) during screening evaluation
Clinically significant abnormalities in hematology or clinical chemistry parameters or Electrocardiogram (ECG), as assessed by the Site Investigator, at the Screening visit that would render the participant unsuitable for participation in the study
Participant's parent or legal guardian is not willing to meet standard of care guidelines (including vaccinations and respiratory syncytial virus prophylaxis if available), nor provide nutritional and respiratory support throughout the study
Clareo Health | A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy
