The commitment of organizations like the Cystic Fibrosis Foundation to global research funding and access initiatives has further propelled market growth. Additionally, major pharmaceutical companies, ...

A thread-like structure in the aspirate was used to prepare smears before ... Among the observed candidate variants, one CFTR gene variant was identified as missense mutations with a likely pathogenic ...

Organoids are three-dimensional structures derived from stem/progenitor cells that can faithfully mimic the intricate structure and physiological function ... Moreover, long-term cultured cells may ...

Boucher, University of North Carolina Cystic Fibrosis-Pulmonary Research and Treatment Center. The cystic fibrosis transmembrane regulator (CFTR) gene was isolated as the cause of cystic fibrosis (CF) ...

The company’s pipeline includes VX-522, a CFTR mRNA therapeutic designed to treat the underlying cause of CF, which is in Phase 1 clinical trial; VX-548, a non-opioid medicine for the treatment ...

A research team has identified the core gene expression networks regulated by key proteins that fundamentally drive phenomena such as cancer development, metastasis, tissue differentiation from stem ...

In living organisms, protein molecules with different chemical structures called proteoforms, are produced from a single gene ...

A research team at KAIST has identified the core gene expression networks regulated by key proteins that fundamentally drive phenomena such as cancer development, metastasis, tissue differentiation ...

The FDA approved a wide range of therapies in 2024, including many new molecular entities and biological products.

Learn the facts on our page, Dispelling Misconceptions About Cystic Fibrosis. In people with CF, mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause the CFTR protein ...

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has approved medicines that treat ...