Cabaletta Bio to Participate in Guggenheim Emerging Outlook
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- 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 ...
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 conference in Milan from June 4-7, 2025. The presentation ...
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 in individuals with cystic fibrosis (CF). Conducted ...
- Deutivacaftor/tezacaftor/vanzacaftor approved for people with cystic fibrosis 6 years and older with at least one responsive mutation in the CFTR gene, including ...
MENLO PARK, Calif.--(BUSINESS WIRE)--ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, today announced that the first patient has been dosed in a ...
Cystic fibrosis represents one of the most common inherited genetic disorders affecting people of European descent, touching the lives of approximately 30,000 individuals in the United States alone. Understanding the genetic foundation of this condition ...
ReCode Therapeutics Initiates Enrollment of Phase 2 Clinical Trial of RCT2100 in Combination with Ivacaftor for the Treatment of Cystic Fibrosis – RCT2100 co-administered with ivacaftor will assess the safety and tolerability of inhaled CFTR mRNA therapy ...
BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that data on TRIKAFTA ® (elexacaftor/tezacaftor/ivacaftor and ivacaftor ...
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) stabilizers, when combined with proprietary CFTR modulators ...