A tiny antibody component could fundamentally transform the treatment of cystic fibrosis: For the first time, researchers have succeeded in developing a so-called nanobody that penetrates directly ...

Sionna Therapeutics today announced the official launch of the company and the closing of a $111 million Series B financing. The round was led by OrbiMed with participation from funds advised by T.

Chemical modulation of histone deacetylase (HDAC) activity by HDAC inhibitors (HDACi) is an increasingly important approach for modifying the etiology of human disease. Loss-of-function diseases arise ...

(Memphis, Tenn.—March 22, 2023) Scientists at St. Jude Children’s Research Hospital and Rockefeller University have combined their expertise to gain a better understanding of the cystic fibrosis ...

A study of 50 adult patients with cystic fibrosis (CF) has found that challenged urine bicarbonate excretion may offer a new, simple, and safe quantification of cystic fibrosis transmembrane ...

Apical localisation of CFTR protein and its chloride channel activity strictly depend on interactions of its PDZ (for postsynaptic density 95/disclarge/zona occludens)-interacting domain in the C ...

Scientists at St. Jude Children's Research Hospital and Rockefeller University have combined their expertise to gain a better understanding of the cystic fibrosis transmembrane conductance regulator ...

UCLA researchers have developed a lipid nanoparticle-based gene-editing approach capable of inserting an entire healthy gene into human airway cells, restoring key biological function in a laboratory ...

An experimental drug suggests that a 'path is clearly achievable' to treat currently untreatable cases of cystic fibrosis disease caused by nonsense mutations. This includes about 11 percent of cystic ...

Alyftrek (vanzacaftor/tezacaftor/deutivacaftor) is a combination medicine approved by the FDA in 2024. Alyftrek is approved to treat people ages 6 and older who have ...

The cystic fibrosis transmembrane conductance regulator has been studied for years but the new efforts have yielded important insights. Scientists at St. Jude Children's Research Hospital and ...