year in review cystic fibrosis modulator therapy efficacy access and evolution

Original title: “Year in Review: Cystic Fibrosis Modulator Therapy-Efficacy, Access, and Evolution.”

lung ultrasound in children with cystic fibrosis a descriptive clinical study

Original title: “LUNG ULTRASOUND IN CHILDREN WITH CYSTIC FIBROSIS: A DESCRIPTIVE CLINICAL STUDY.”

Cystic fibrosis (CF) is a genetic disorder that causes problems with breathing and digestion.

Original title: “About Cystic Fibrosis”

Sionna’s medicine didn’t have a meaningful additive impact when combined with Vertex’s top-selling Trikafta, surprising analysts and collapsing company shares.

Original title: “Sionna cystic fibrosis drug fails key trial, removing threat to Vertex”

A surprise flop in Phase 2a cystic fibrosis trial sent Sionna’s shares plummeting, but for Vertex, the news reinforced its standard-of-care standing with highly successful drugs like Trikafta.

Original title: “Sionna’s cystic fibrosis failure is a windfall for Vertex’s blockbuster franchise”

cystic fibrosis associated amyloid a amyloidosis a rare but devastating complication with poor prognosis

Original title: “Cystic fibrosis-associated amyloid a amyloidosis: a rare but devastating complication with poor prognosis.”

Despite another strong earnings report, analysts warned that upcoming cystic fibrosis data from Sionna Therapeutics "looms large" over Vertex.

Original title: “Vertex’s biggest overhang: a rival 50 times smaller”

The deal adds to a growing streak of partnerships involving new types of T cell engagers and gives Vertex another chance to broaden a business still reliant on cystic fibrosis medicines.

Original title: “AbCellera, Vertex partner on next-gen T-cell engagers”

clinical and biochemical response to elexacaftor tezacaftor ivacaftor in a patient with the rare l927p n1303k cftr genotype

Original title: “Clinical and biochemical response to elexacaftor/tezacaftor/ivacaftor in a patient with the rare L927P/N1303K CFTR genotype.”

profiling the cftr variant selectivity and off target interactions of vx 121

Original title: “Profiling the CFTR Variant Selectivity and Off-Target Interactions of VX-121.”

economic evaluation of cftr modulator therapies in cystic fibrosis a cost benefit and cost effectiveness perspective from the remedia european commission project

Original title: “Economic evaluation of CFTR modulator therapies in cystic fibrosis: a cost-benefit and cost-effectiveness perspective from the REMEDIA European commission project.”

clinical profile of cystic fibrosis in india a multi centric prospective study

Original title: “Clinical Profile of Cystic Fibrosis in India: A Multi-Centric Prospective Study.”

ecfs statement on theratyping and theranostics in the context of rare and ultrarare cftr variants in people with cf

Original title: “ECFS statement on theratyping and theranostics in the context of rare and ultrarare CFTR variants in people with CF.”

An FDA initiative to implement real-world clinical trials could dramatically accelerate the pace at which new treatments become available to patients, according to physicians from two institutions involved in the effort’s initial phase.The approach will leverage electronic health records ... An FDA initiative to implement real-world clinical trials could dramatically accelerate the pace at which new treatments become available to patients, according to physicians from two institutions involved in the effort’s initial phase.The approach will leverage electronic health records and an AI-powered platform to allow FDA scientists to track safety signals and efficacy endpoints while Makary called the initiative “a critical step” in the agency’s broader goal to facilitate continuous trials and move away from the traditional clinical development model that consists of multiple distinct phases — each with its own protocol, a separate study and lag time in between. Two proof-of-concept real-time trials — both of which are evaluating oncology therapies — got underway this month. The randomized phase 2 TRAVERSE study is evaluating acalabrutinib (Calquence, AstraZeneca) in combination with venetoclax (Venclexta; AbbVie, Genentech) and rituximab for treatment-naive mantle cell lymphoma. This could open the door for application of data science to multiple different aspects of how we evaluate novel therapies, which ideally — in aggregate — could result in speeding up the process.” · Jennifer Keating Litton, MD, MHCM, can be reached at [email protected]. Emma A. Meagher, MD, can be reached at [email protected]. ... FDA announces major steps to implement real-time clinical trials. We need our clinical trials to evolve at the same pace that scientific breakthroughs are happening,” Jennifer Keating Litton, MD, MHCM, chief clinical research officer and professor in the department of breast medical oncology at The University of Texas MD Anderson Cancer Center, told Healio.

Original title: “Real-time clinical trials may ‘fundamentally transform’ research landscape”