AR-501, an investigational inhaled formulation of gallium citrate to treat chronic lung infections in patients with cystic fibrosis (CF), has been granted orphan drug status by the U.S. Food and Drug Administration (FDA). The treatment, developed by Aridis Pharmaceuticals, is designed to be given once weekly…
Children often mistakenly pronounce cystic fibrosis (CF) as “65 roses.” Eilís Moroney and Ruth Cahill, both parents of children with this progressive lung disease, have turned that endearing practice into a book aimed at explaining CF to youngsters in an age-appropriate way. Titled “Our Baby has Sixty-Five Roses,”…
Ultrasound imaging can be a reliable diagnostic tool for early detection of liver disease in children with cystic fibrosis (CF), a study finds. Using this strategy can help clinicians identify CF patients most at risk for liver damage caused by cirrhosis (tissue scarring), researchers suggest. These findings were reported in…
More than 60 cystic fibrosis experts strongly support Relizorb being made available to CF patients on tube feeding, pushing in a commentary for the insurance coverage that’s generally denied this digestive enzyme cartridge, and inviting “the CF community” to become advocates of better patient access to the device. Their opinion…
A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet syndrome were among winners of the 2019 Rare Impact Awards. Officials of the National Organization for Rare Disorders (NORD) presented the awards during a June 22 dinner attended by…
The U.S. Food and Drug Administration (FDA) has approved Symdeko (tezacaftor/ivacaftor and ivacaftor) tablets for the treatment of children, ages 6 to 11 years, with cystic fibrosis (CF) who have certain mutations that respond to treatment, its developer, Vertex Pharmaceuticals, has announced. In order…
Long-term use of inhaled antibiotics, given to treat lung infections caused by the bacteria Pseudomonas aeruginosa in people with cystic fibrosis (CF), can for young children raise the risk of a new infection by Aspergillus fungi, a study suggests. The study, “Pseudomonas…
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Treatment with Orkambi significantly improves exercise tolerance in adult cystic fibrosis patients with severe disease as early as 4 weeks after treatment — as shown by results of the six-minute walk test (6MWT), a study found. The findings support the use of exercise tolerance as an important…
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