Spirovant Sciences‘ lead gene therapy candidate for select cystic fibrosis (CF) patients, SPIRO-2101, was given rare pediatric disease and orphan drug designations by the U.S. Food and Drug Administration (FDA) to support its development, according to a recent press release. Orphan drug designation applies to medicines targeting…
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The U.S. Food and Drug Administration (FDA) has approved Vertex Pharmaceuticals‘ Kalydeco (ivacaftor) as a treatment for infants as young as four months with cystic fibrosis (CF) caused by certain mutations. The approval makes Kalydeco the first medication in its class to be approved for…
Nonprofit Emily’s Entourage has awarded a total of $790,000 to four research projects focused on potential therapies for cystic fibrosis (CF) and related lung infections. To be given over two years, the four grants “focus on two critical unmet needs for the CF community: therapies that address nonsense…
Gastrointestinal (GI) problems are common to people with cystic fibrosis (CF), especially those with pancreatic insufficiency, and can considerably disrupt daily life, an online survey by researchers in the U.K. reported. More attention needs to be given to the disease’s GI complications, the research team said. CF is primarily…
The public is being invited to vote on 2020 AbbVie Scholarships for students with cystic fibrosis (CF). Voting is now open to award two meritorious students with scholarships totaling $25,000 each. Until Sept. 30 (at 11 a.m. ET), the public may visit the official AbbVie CF Scholarship website…
The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended extending the licenses for Symkevi and Kalydeco, two cystic fibrosis (CF) treatments marketed by Vertex Pharmaceuticals, to allow these medications to be used in younger children. If approved by…
Moderna Therapeutics and Vertex Pharmaceuticals have entered a three-year research and licensing agreement aimed at developing gene-editing delivery techniques for the treatment of cystic fibrosis (CF). While Moderna will focus on ways to deliver gene-editing therapies to lung cells, Vertex will concentrate on other gene-editing components —…
The 7th edition of Bike 2 Breathe, an initiative aiming to raise funds and awareness for cystic fibrosis (CF), is planned to start on Sept. 21 in Boston. The event was created by Jerry Cahill, a CF patient and ambassador for the Boomer Esiason…
Adding pancreatic enzymes to “pre-digest” food was found to be an effective way to ensure nutrition in an infant with cystic fibrosis who was being fed through a feeding tube, according to a recent case report. The report, “Crushing pancreatic enzymes with enteral feeds in an extremely…
The European Medicines Agency (EMA) has validated an application that seeks to expand the approval of Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination with Kalydeco (ivacaftor) to treat cystic fibrosis (CF) in individuals 12 and older who have at least one copy of the F508del mutation…
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