The U.S. Food and Drug Administration (FDA) approved the use of ivacaftor (Kalydeco®) today for people ages 2 and older with cystic fibrosis who have at least one of the following five splice mutations: 3849+10kbC->T, 2789+5G->A, 3272-26A->G, 711+3A->G, E831X.
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The U.S. Food and Drug Administration (FDA) today approved ivacaftor (Kalydeco®) for people ages 2 and older who have at least one of 23 residual function mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Today, the Cystic Fibrosis Foundation announced that it will invest up to $8.4 million in SpliSense's Series B funding round to develop an antisense oligonucleotide therapy for people with cystic fibrosis who have splicing mutations and potentially other rare mutations.
The studies aim to improve early detection of chronic lung allograft dysfunction (CLAD), the leading cause of lung transplant failures.
The patient-centered outcomes research (PCOR) training manual provides discrete steps, tools, and resources that CF Center research teams can take to integrate and maintain patient/caregiver-partners in CF research.
New funding awards include up to $2.6M to Eloxx Pharmaceuticals to identify potential therapies for CF nonsense mutations
The intent of this request for applications is to solicit and fund projects that will improve our understanding of the biological basis for the development and progression of gastrointestinal manifestations in cystic fibrosis as well as to identify potential novel therapeutic strategies to manage and treat them.
Clinical postdoctoral research fellowships are offered to support postdoctoral research training related to cystic fibrosis. These awards are intended to enable training in new research areas and methods to advance the scientific knowledge of the applicant and to collect data to enable their transition into an impactful research career.