7 CFTR Articles You Can’t Miss
1. Partnership to Promote Next-Generation Diagnostic Test for CF
Read about a partnership to simplify the adoption of next-generation DNA sequencing for routine diagnostic laboratories. According to the terms of the agreement, Sophia Genetics will use its advanced analytical platform, Sophia DDM, to support Devyser’s NGS diagnostic kits for cystic fibrosis and hereditary breast cancer.
2. CFTR Abundant in Gut, Possible Reason for Intestinal Problems in CF Patients
The cystic fibrosis transmembrane conductance regulator (CFTR), a key protein that is defective in people with cystic fibrosis, is abundant in the gastrointestinal tract of people and may be a reason why CF patients are troubled by gastrointestinal problems, a recent collaborative study reported. Read the article here.
3. Data on Inhaled CF Therapy in Clinical Testing To Be Presented at NACFC 2016
ProQR Therapeutics will announce proof-of-concept results from tests on its lead candidate to treat cystic fibrosis, QR-010, at a scientific conference this year in October. QR-010 is a first-in-class RNA-based oligonucleotide aimed to treat CF patients who carry the common ∆F508 mutation, which affects more than 70% of all patients. Read the article here.
4. Vertex Enrolling Patients with CF in Three, Phase 3 Studies of VX-661 and Kalydeco Combo Therapy
Vertex Pharmaceuticals recently gave an update on its Phase 3 development program, which consists of four studies, on the investigational compound VX-661 in combination with Kalydeco (ivacaftor) as a treatment of patients with cystic fibrosis. Read the article here.
5. CF Patients Being Recruited for Phase 1 Study of Oral CFTR Amplifier
Proteostasis Therapeutics announced the enrollement of cystic fibrosis patients and healthy adults in two Phase 1 clinical trials evaluating its lead drug candidate PTI-428, an oral amplifier of the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
6. Full Gene Sequencing Might Detect CF Mutations in Babies of Any Ethnicity, Study Says
Babies with a mutation known to cause cystic fibrosis and a second mutation called the 5T allele should be screened for additional mutations to predict their risk of developing CF later in life, according to a study. Read about it here.
7. CF Foundation Research Conference Gathers Genetic Experts From Many Fields
The Cystic Fibrosis Foundation hosted more than 100 researchers from various fields at its “New Technologies Advancing Toward a One-time Cure” conference last June to debate how new research and treatments for genetic diseases across the board could lead to a cure for cystic fibrosis. Read about it here.
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