Health Canada has expanded the approval of Vertex Pharmaceuticals‘ triple-combination modulator treatment Trikafta to cover children with cystic fibrosis (CF) as young as 6 who have at least one copy of the F508del mutation. Trikafta had been approved only for patients ages 12 and older in Canada. According to…
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A fungus called Aspergillus fumigatus can significantly affect lung function and structure, as well as increase the number of pulmonary exacerbations — episodes of a sudden worsening of respiratory symptoms — in children with cystic fibrosis (CF), a study suggests. The findings shed light on the need for early…
Sionna Therapeutics, a life sciences company that is developing a pipeline of experimental treatments for cystic fibrosis (CF), has announced its official launch following the closing of a $111 million Series B financing. The company is aiming to treat the underlying cause of CF. “Our mission at Sionna is…
The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…
As part of its Path to a Cure initiative, the Cystic Fibrosis Foundation is readying its first “Golden Ticket” Competition, which aims to support early research toward new treatments for cystic fibrosis (CF). The competition will open on May 2, and applications are being accepted through May 30. It…
Marking three decades of supporting the educational pursuits of young cystic fibrosis (CF) patients, AbbVie is calling for applications from U.S. students in this, its 30th year offering CF scholarships. The application deadline for the AbbVie CF Scholarship program for the 2022-2023 academic year is May 25 at…
Difficulties with eating and proper nutrition for cystic fibrosis (CF) patients may be due to an inflammation of the esophagus known as eosinophilic esophagitis (EoE), according to a report on three young children in the U.S. Shared traits, particularly gastrointestinal issues like gastroesophageal reflux disease (GERD) and abdominal pain that leave children…
Triple combination therapies, a mix of CFTR correctors and potentiators, significantly improve lung function, reduce sweat chloride, and enhance the quality of life for people with cystic fibrosis (CF), a meta-analysis of pooled results from multiple clinical trials shows. The meta-analysis, “Efficacy and Safety of Triple…
The first participant has been enrolled in a Phase 1 clinical trial testing the safety and pharmacological properties of the antibiotic Xenleta (lefamulin) in adults with cystic fibrosis (CF). “We are excited to commence enrollment in this important study to evaluate the potential of Xenleta in the management…
The National Organization for Rare Disorders (NORD) has updated its State Report Card to make it more digitally friendly and added telehealth to its categories of rare disease policy issues in a nod to its increased use during the ongoing COVID-19 pandemic. NORD’s report card project began seven…
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