4 results
Primary: To evaluate the efficacy of once-weekly lonapegsomatropin compared to placebo at 38 weeks in adults with growth hormone deficiency (GHD). Secondary: 1. To evaluate the safety and tolerability of once-weekly lonapegsomatropin in adults with…
This study will evaluate the safety, pharmacokinetics, pharmacodynamics, and efficacy of crovalimab compared with placebo for the management of acute uncomplicated vaso-occlusive episodes (VOE) in patients with sickle cell disease (SCD).
This study will evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of crovalimab compared with placebo as adjunct treatment for the prevention of vaso-occlusive episodes (VOEs) in patients with sickle cell disease (SCD).
The objective of the study is to assess the long-term safety and efficacy of intravenous ATB200 co-administration with oral AT2221 in adult subjects with late-onset Pompe disease.