Cutting-Edge Cystic Fibrosis Therapies Are Overpriced. This Campaign is Calling for Greater Access
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Cutting-Edge Cystic Fibrosis Therapies Are Overpriced. This Campaign is Calling for Greater Access

In recent years, treatment options for cystic fibrosis, a rare disease, have increased significantly in number and in effectiveness. However, many of the most effective treatments, developed by Vertex Pharmaceuticals,…

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Newborn Screenings Miss Cystic Fibrosis in Non-White Newborns
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Newborn Screenings Miss Cystic Fibrosis in Non-White Newborns

Newborn screening is a public health program in which infants are screened for various metabolic, genetic, and developmental disorders shortly after birth. Through newborn screening, doctors may identify potential disorders…

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SPL84-23-1 for CF Earns Orphan Drug Status
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SPL84-23-1 for CF Earns Orphan Drug Status

In a news release from January 4, 2022, biopharmaceutical company SpliSense shared that their drug candidate SPL84-23-1 for cystic fibrosis (CF) earned Orphan Drug designation in both the United States…

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ICYMI: Cystic Fibrosis Drug TRIKAFTA Accepted for Priority Review in Canada for Patients Aged 6-11
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ICYMI: Cystic Fibrosis Drug TRIKAFTA Accepted for Priority Review in Canada for Patients Aged 6-11

  In June 2021, Health Canada approved TRIKAFTA (elexacaftor/tezacaftor/ivacaftor) for patients with cystic fibrosis (CF), with at least one F508del mutation, aged 12+. According to a news release from late…

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The Unusual Clinical Profile of Three Brothers With Cystic Fibrosis Warrants a New Look at Standard CF Care
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The Unusual Clinical Profile of Three Brothers With Cystic Fibrosis Warrants a New Look at Standard CF Care

  Science Direct recently published a Respiratory Medicine Case Report that may impact future Cystic Fibrosis (CF) treatment. The case involves adult brothers herein referred to for purposes of this…

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FRC Test Detects CF in Newborn Screening
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FRC Test Detects CF in Newborn Screening

Have you ever heard of newborn screening? Basically, newborn screening is a public health service which can identify potential hormone-related, genetic, or metabolic conditions. Since early identification is so crucial…

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UK Cystic Fibrosis Influencer Marc Cotterill Presents ‘Patient View’ Video at European Cystic Fibrosis Conference

Ahead of Cystic Fibrosis Week in the UK, 37-year-old Marc Cotterill, who lives with the disease, has given a video presentation to the European Cystic Fibrosis Conference on the theme…

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TRIKAFTA Approved for Pediatric Patients with F508del-Mutated Cystic Fibrosis
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TRIKAFTA Approved for Pediatric Patients with F508del-Mutated Cystic Fibrosis

In a news release from June 9, 2021, biotechnology company Vertex Pharmaceuticals, Inc. ("Vertex") shared that its therapy TRIKAFTA (elexacaftor/texacaftor/ivacaftor and ivacaftor) was approved for expanded use. Now, the treatment…

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