Learn about the research studies we conduct with commercial and academic partners, and the clinical trials we support for pharmaceutical sponsors.
Ornithine transcarbamylase (OTC) deficiency clinical research study
This study is a randomized, placebo-controlled study, testing an investigational medicine called ARCT-810 on adults with ornithine transcarbamylase (OTC) deficiency.
Anti-AChR antibody-positive generalized myasthenia gravis clinical research study
This placebo-controlled Phase 3 study is testing an investigational medicine called ravulizumab-cwvz, also known in this study as ALXN1210, on adults with anti-AChR antibody-positive, generalized myasthenia gravis (gMG).
What do you think about your cystic fibrosis treatment(s)?
This study aims to learn what people with cystic fibrosis actually think about taking the CFTR modulator therapies Kalydeco, Orkambi, Symdeko/Symkevi, and Trikafta/Kaftrio. This is not like other studies that may only focus on following doctors’ orders.
What do you think about your sickle cell treatment(s)?
This study aims to learn what people with sickle cell actually think about taking the newer sickle cell medications – Adakveo (crizanlizumab), Endari (L-glutamine), and Oxbryta (voxelotor) – and see how they compare with the older drug, hydroxyurea. This is not like other studies that may only focus on following doctors’ orders.