Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a ways to go in the United States. No state today tests for all 35 disorders recommended under a federal screening panel, and even in those that come close, rare…
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People using a nebulizer while lying on their side showed no significant loss in a treatment’s overall deposition in the lungs, a study in healthy adults and in cystic fibrosis (CF) patients reports. Rather, it found that alternating sides instead of sitting upright may better deposit a medicine’s active compounds…
Employees at the Development Bank of Wales, in the U.K., have raised £30,400 (around $37,000) to support the Cystic Fibrosis Trust. Every year, bank employees are asked to nominate a charitable organization to support, with which they have a personal connection. This year, the Cystic Fibrosis Trust was…
The first patient has been dosed in a Phase 2 clinical trial testing Proteostasis Therapeutics‘s combinations of three investigational therapies for cystic fibrosis (CF), the company announced. The three treatments under investigation are all designed to fix the defective CFTR protein due to mutations in the CFTR gene, which is the…
Jeffrey Leiden, who in seven years has taken Vertex Pharmaceuticals to the cusp of developing treatments for about 90% of people with cystic fibrosis (CF), is exchanging his day-to-day leadership role as CEO for an elder statesman position. Leiden — who finished high school at 15, earned…
Five programs helping to empower the cystic fibrosis (CF) community were selected to receive up to $10,000 each under the fourth annual Impact Grants awarded by the Cystic Fibrosis Foundation (CFF). Launched in 2016, the Impact Grants program is designed to support new ideas and projects that aim…
The European Commission (EC) and the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) granted Mylan the right to market TOBI Podhaler (tobramycin inhalation powder) and TOBI Solution (tobramycin nebulizer solution) in the United Kingdom, the company announced. The decision came after the Committee…
The European Medicines Agency (EMA) granted orphan drug status to AR-501, a treatment candidate for lung infections in people with cystic fibrosis (CF). AR-501, developed by Aridis Pharmaceuticals and also known as Panaecin, is an inhaled formulation of gallium citrate intended as a weekly and self-administered therapy delivered…
Vertex Pharmaceuticals is asking the U.S. Food and Drug Administration to approve a first triple combination therapy — elexacaftor (VX-445) plus tezacaftor, and ivacaftor (Kalydeco) — to treat cystic fibrosis (CF) patients who cannot use its other disease-modifying treatments or don’t benefit as intended. Triple combinations include…
Treatment with Kalydeco (ivacaftor) improved lung function, exercise capacity, and sweat chloride concentration in cystic fibrosis (CF) patients with severe lung disease, a small retrospective analysis shows. The study, “Effectiveness of ivacaftor in severe cystic fibrosis patients and non‐G551D gating mutations,” was published in the journal Pediatric Pulmonology. Mutations…
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