CFTR

Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015-2022): 2022 interim analysis

38 views | Oct 11 2023

The interim findings of the STRIDE registry show that long-term treatment with ataluren plus standard of care delays disease progression milestones in individuals with nonsense mutation Duchenne muscular dystrophy (nmDMD) compared to standard of care alone. [Read the Full Post]

Positive and negative impacts of elexacaftor/tezacaftor/ivacaftor: Healthcare providers' observations across US centers

32 views | Sep 02 2023

This study found that while Elexacaftor/tezacaftor/ivacaftor (ETI) had positive physical effects in cystic fibrosis patients, it was also associated with negative side effects such as sleep difficulties and worsening psychological functioning. [Read the Full Post]

Real-world outcomes and direct care cost before and after elexacaftor/tezacaftor/ivacaftor initiation in commercially insured members with cystic fibrosis

25 views | Sep 02 2023

The study found that among commercially insured individuals with cystic fibrosis initiating treatment with ELX/TEZ/IVA, there was a significant increase in total cost of care, primarily driven by increased pharmacy benefit costs, despite notable reductions in healthcare resource utilization and pulmonary exacerbation events. [Read the Full Post]

Fueling the Pipeline via Innovations in Organic Synthesis

16 views | Aug 10 2023

Synthetic organic chemistry, particularly through the involvement of Discovery Synthesis Groups like AbbVie's Centralized Organic Synthesis (COS), is of paramount importance in the pharmaceutical industry as it accelerates the drug discovery process, addresses synthetic accessibility challenges, and contributes to the development of advanced drug candidates in various therapeutic areas. [Read the Full Post]

Novel Correctors and Potentiators Enhance Translational Readthrough in CFTR Nonsense Mutations

33 views | Aug 10 2023

The study evaluated the effectiveness of novel CFTR correctors, potentiators, and readthrough compounds in restoring CFTR function in cells with premature-termination codon (PTC) mutations, showing promising results for the potential treatment of cystic fibrosis. [Read the Full Post]

CFTR modulates aquaporin-mediated glycerol permeability in mouse Sertoli cells

26 views | May 24 2023

This study explores the potential molecular partnership between CFTR and AQPs in mouse Sertoli cells and suggests that the malfunction of CFTR and the consequent alteration in glycerol permeability is a potential link between male infertility and cystic fibrosis. [Read the Full Post]

Eradication of Mycobacterium abscessus infection in cystic fibrosis with initiation of Elexacaftor/Tezacaftor/Ivacaftor

24 views | May 19 2023

This case highlights the potential positive impact of ETI, a CFTR modulator combination, on the treatment of challenging infections such as M. abscessus in CF. [Read the Full Post]

The Impact of N-nitrosamine Impurities on Clinical Drug Development

83 views | Feb 26 2023

The presence of N-nitrosamine impurities in commercially available drugs has led to shortages of marketed drugs and complications in the clinical development of new investigational products, with alternative approaches being sought for commonly employed perpetrators in DDI studies. [Read the Full Post]

Mutual Effects of Single and Combined CFTR Modulators and Bacterial Infection in Cystic Fibrosis

23 views | Jan 28 2023

Cristina Cigana et al. suggested optimizing host- and pathogen-directed therapies to improve efficacy for personalized treatment. [Read the Full Post]

Treatment effects of Elexacaftor/Tezacaftor/Ivacaftor in people with CF carrying non-F508del mutations

33 views | Nov 18 2022

Galit Livnat et al. demonstrated the clinical efficacy of ETI in pwCF carrying CFTR processing non-F508del mutations which were predicted to respond by in vitro studies. [Read the Full Post]