- ALYFTREK™ is approved for patients 6 years and older with at least one responsive mutation, including 31 additional mutations not responsive to other CFTR modulator therapies - - In head-to-head ...
The cost of medication was too high for thousands of CF sufferers around the world. Now a Bangladeshi company is making a generic version that will change lives ...
Arcturus Therapeutics Holdings Inc. (ARCT) is gearing up to report results from its phase 1b study using its inhaled messenger RNA [mRNA] treatment known as ARCT-032 for the treatment of patients with ...
Hundreds of people with cystic fibrosis are to be offered a new pill which has been hailed as “life changing” by health experts. CF is caused by a faulty gene that affects the production of a protein ...
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 ...
DENVER - A recent retrospective study published in CHEST(Opens in a new window) evaluated the effects of cystic fibrosis transmembrane conductance regulator (CFTR) modulator use on pregnancy outcomes ...
- Data presented on outcomes following treatment with CFTR modulators add to growing body of evidence that reduced level of sweat chloride is associated with improved clinical outcomes, particularly ...
The Medicines and Healthcare products Regulatory Agency (MHRA) has extended the licence of the cystic fibrosis medicines Kaftrio (ivacaftor, tezacaftor, and elexacaftor) and Kalydeco (ivacaftor) for ...
4D Molecular Therapeutics (4DMT) is pressing ahead with evaluation of its gene therapy, 4D-710, in a Phase II dose expansion trial in cystic fibrosis (CF) after the drug displayed positive ...
Sionna Therapeutics offers a high-risk, high-reward play, with upside tied to clinical success of its NBD1 stabilizers and CFTR modulators for cystic fibrosis. Strong cash position ($260 million+) and ...
Could next-generation gene editing mean a cure for cystic fibrosis (CF)? There's been a lot of advancement on the disease in the last decade, but there are still those living with the disease who get ...
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