Vertex Pharmaceuticals Incorporated

This small, early-stage study tested an oral drug called VX-147 in 16 adults with a specific genetic form of kidney disease called APOL1-mediated FSGS. The goal was to see if the drug could reduce the amount of protein leaking into the urine, which is a key sign of kidney damage.…

This study tested how a triple-combination drug (elexacaftor/tezacaftor/ivacaftor) affects daily cough frequency and physical activity in people with cystic fibrosis. It involved 82 participants aged 12 and older with a specific genetic profile. Researchers used wearable sensors …

This study tested a one-time treatment using the gene-editing tool CRISPR to try to help people with severe thalassemia. Doctors took a patient's own blood stem cells, edited a gene in the lab, and then gave them back. The main goal was to see if this single treatment could allow…

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 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 how well the medication suzetrigine works to control pain after common surgeries like laparoscopic procedures and arthroscopic joint surgeries. Researchers enrolled 47 people who were scheduled for these procedures to see if the drug effectively reduced their po…

This study tested whether an investigational drug called VX-548 could safely reduce pain in adults with chronic leg pain caused by a pinched nerve in the lower back (a condition called lumbosacral radiculopathy). 218 participants were randomly assigned to receive either VX-548 or…

This study tested how well and how safely a drug called suzetrigine works to reduce pain when used as part of a standard pain management plan. It involved 100 adults who had cosmetic or reconstructive surgery and would normally need strong pain medication afterward. Researchers m…

This early-stage study aimed to see if an experimental drug for polycystic kidney disease (VX-407) changes how the body processes common birth control pills. Researchers tested this in 74 healthy volunteers by giving them both the new drug and different types of oral contraceptiv…

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…