Participants were randomly assigned to receive Trikafta or placebo based on age and weight: Aged 6 to < 12 years and less than 30 kg: morning dose of elexacaftor 100 mg/tezacaftor 50 mg/ivacaftor ...

Product approvals in regenerative medicine and rare diseases are among the highlights in our recap of recent FDA regulatory decisions.

Enzyme mechanisms are the chemical transformations, and the steps within them, generated by enzymatic action on substrates. The mechanism of enzyme catalysis is similar in principle to other types ...

From Drug Discovery Biology, Monash Institute of Pharmaceutical Sciences, Monash University, Parkville Campus, Victoria, Australia (R.H.R.) Division of Endocrinology and Metabolism (E.D.A.), ...

NHS England has agreed a deal with Vertex for its three-drug cystic fibrosis therapy Kaftrio, just days after the EMA’s human medicines committee recommended approval of the drug. The deal means ...

Sales are expected to surpass that total in 2024. Like Vertex’s top-selling drug Trikafta, Alyftrek is a three-drug combination that uses a compound called tezacaftor as its chemical backbone. The two ...