Healio

‘CFTR dysfunction may be a lot more common’ in healthy young adults

Please provide your email address to receive an email when new articles are posted on . Sweat chloride values ranging from 30 mEq/L to 59 mEq/L were observed in “a surprisingly high proportion of ...
Healio

Aerosol gene therapy safe, heightens CFTR protein levels in cystic fibrosis

Please provide your email address to receive an email when new articles are posted on . Patients given aerosolized 4D-710 had elevated CFTR protein levels that went over normal levels. Over 12 months, ...
Kaleido Scope

Gene Expression Core

The Gene Expression Core was founded in 1994 and promotes translational, multidisciplinary CF research by furnishing expertise concerning recombinant CFTR and other proteins relevant to CF disease ...
Nature

A large deletion in the CFTR gene in CBAVD

Purpose: Most cystic fibrosis mutation screening methods do not detect large exon deletions or duplications in the cystic fibrosis transmembrane regulator gene. We looked for such mutations in ...
Yahoo Finance

Vertex Announces European Commission Approval of ALYFTREK®, a New Once-Daily CFTR Modulator for the Treatment of Cystic Fibrosis

- ALYFTREK ® (deutivacaftor/tezacaftor/vanzacaftor) approved in the EU for people with CF 6 years and older with at least one non-class I mutation in the CFTR gene ...
Yahoo Finance

Vertex Announces UK MHRA Approval of ALYFTREK® (Deutivacaftor/Tezacaftor/Vanzacaftor), a Once-Daily Next-in-Class CFTR Modulator for the Treatment of Cystic Fibrosis

- Deutivacaftor/tezacaftor/vanzacaftor approved for people with cystic fibrosis 6 years and older with at least one responsive mutation in the CFTR gene, including ...
Nasdaq

Sionna Therapeutics Presents Preclinical Data on Novel CFTR Modulators at the 48th European Cystic Fibrosis Conference

Sionna Therapeutics, Inc. has announced the presentation of promising preclinical data at the 48th European Cystic Fibrosis Conference in Milan, showing that its nucleotide-binding domain 1 (NBD1) ...
News Medical

CFTR modulator can be safe, well-tolerated for younger children with common form of cystic fibrosis

Children ages two to five who have the most common form of cystic fibrosis (CF), caused by two copies of the F508 gene mutation, have not had any modulator treatments available to them until recently.
Kaleido Scope

A small molecule induces readthrough of cystic fibrosis CFTR nonsense mutations

The drug is a small molecule with a novel mechanism of action, say David Bedwell, Ph.D., and Steven Rowe, M.D., MSPH, co-senior authors. Bedwell is professor and chair of the University of Alabama at ...
Medindia

Genetics of Male infertility - CFTR Gene Mutations

Mutations in the cystic fibrosis trans membrane conductance regulator gene cause congenital bilateral absence of the vas deferens (CBAVD) in approximately 1% of the infertile males. It is a common ...
Nasdaq

Sionna Therapeutics to Present Preclinical Data on CFTR Modulators at European Cystic Fibrosis Conference

Sionna Therapeutics, Inc. announced that preclinical data on its novel cystic fibrosis (CF) treatment candidates, SION-451 and SION-719, will be presented at the European Cystic Fibrosis Society’s ...