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) ...
Please provide your email address to receive an email when new articles are posted on . Triple therapy improved lung function, BMI and other factors. Patients naïve to cystic fibrosis transmembrane ...
BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration has cleared its Investigational New Drug (IND) application for VX ...
- 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 ...
Please provide your email address to receive an email when new articles are posted on . “[These findings] help clinicians be more supportive of a female with CF becoming pregnant,” Raksha Jain, MD, ...
BETHESDA, Md.--(BUSINESS WIRE)--The Cystic Fibrosis Foundation today announced its investment of $5 million in Sionna Therapeutics, a company founded in 2019. The company is focused exclusively on ...
PITTSBURGH, Jan. 08, 2026 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS) today announced a positive interim clinical update from the highest dose cohort of CORAL-1, the ...
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 ...
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 ...
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 ...
Confirmed wild-type CFTR delivery and expression in conducting airway cells of patients with class I mutations KB407 transduction confirmed in all six patients with successful bronchoscopies ...
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