Vertex Pharmaceuticals Incorporated

This study tested a one-time treatment for people with severe beta-thalassemia, a genetic blood disorder that requires regular blood transfusions to survive. Doctors used CRISPR gene-editing technology to modify a patient's own blood stem cells, then gave them back as a transplan…

This study tested how well a three-drug combination (elexacaftor/tezacaftor/ivacaftor) works for people with cystic fibrosis. It specifically measured if the treatment could reduce daily coughing and increase physical activity in patients aged 12 and older. Researchers used weara…

This study followed cystic fibrosis patients aged 2 and older to see how safe and effective a three-drug combination therapy is over the long term. Participants had previously taken these medications in an earlier study and continued treatment to monitor their response. The resea…

This study tested a three-drug combination therapy (elexacaftor/tezacaftor/ivacaftor) in very young children with cystic fibrosis, aged 12 to 24 months. The main goals were to see how the medicine behaves in the body and to check if it is safe and well-tolerated in this age group…

This early-stage study tested a new granule form of a cystic fibrosis drug combination in healthy adults. Researchers wanted to see how the body absorbs the granules, if taking them with food makes a difference, and if different doses work as expected. The main goal was to gather…

This study followed 507 people with cystic fibrosis to check the long-term safety and effectiveness of a three-drug combination therapy. It focused on patients with a specific genetic mutation (F508del) that causes most CF cases. Researchers monitored lung function, weight, quali…

This study tested an experimental drug called VX-147 in adults with a specific genetic form of kidney disease called APOL1-mediated FSGS. The trial involved 16 participants with confirmed genetic markers and kidney biopsy diagnosis. Researchers measured how well the drug reduced …

This study tested the safety and effectiveness of a two-drug combination (VX-661 and ivacaftor) for people with cystic fibrosis who have two copies of the most common genetic mutation (F508del). Forty participants were randomly assigned to receive either the drug combination or a…

This study tested whether the medication VX-548 could safely reduce leg pain caused by a pinched nerve in the lower back (lumbosacral radiculopathy). 218 adults with this chronic pain condition for over 3 months were randomly assigned to receive either VX-548 or a placebo. Resear…

This study tested how well and how safely the drug suzetrigine worked when used as part of a multi-drug plan to manage pain after cosmetic or reconstructive surgery. It involved 100 adults who were expected to need strong pain medication for at least 3 days after their procedure.…

This study tested whether suzetrigine helps control pain after laparoscopic abdominal surgery or arthroscopic joint surgery. Researchers enrolled 47 patients who would normally need opioid pain medication after these procedures. They measured how well the drug worked and checked …

This early-stage study tested how the body absorbs different versions of the experimental drug povetacicept. It involved 136 healthy volunteers to compare two drug formulations and presentations. The main goal was to see if different forms of the drug behave similarly in the body…

This study aimed to understand how cystic fibrosis affects the pancreas in babies during their first year of life. Researchers observed 79 infants who were not taking certain CF-specific medications. They measured a substance in stool samples over time to see how well the pancrea…