With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Creditand affordable health insurance to future funding for rare disease research. Yet “whether Democrats take over the House or Senate, or…
News
Once-daily acebilustat, an investigational oral therapy for cystic fibrosis (CF) under development by Celtaxsys, reduces the frequency and delays the onset of pulmonary exacerbations in CF patients, results from a Phase 2 study show. Patients with better lung function…
Treatment with lenabasum can effectively prevent inflammation and induce pro-resolving signals in airway immune cells from patients with cystic fibrosis (CF), results from a preclinical study show. Lenabasum’s more recent preclinical data was discussed at the 32nd North American Cystic Fibrosis Conference, in a poster presentation titled “…
Vertex Pharmaceuticals’ VX-659 or VX-445 in combination with Kalydeco (ivacaftor) and tezacaftor (VX-661) can improve the lung function of patients with cystic fibrosis (CF) with one or two copies of the CFTR F508del mutation, Phase 2 data shows. Updated results of the studies were discussed…
Treatment with a combo of Proteostasis Therapeutics’ investigational CFTR modulator therapies — PTI-808, a potentiator, plus PTI-801, a corrector — can promote significant improvements in lung function and reduction of sweat chloride in patients with cystic fibrosis (CF), Phase 1 data show. So far in the Phase…
Santhera Pharmaceuticals’ investigative inhalation therapy for cystic fibrosis (CF), POL6014, has been favored for orphan drug designation within the European Union. A recommendation supporting the designation, which offers financial and regulatory incentives to develop treatments for rare diseases, was made by …
AzurRx BioPharma is planning a multi-center Phase 2 trial to be conducted in the United States and Europe to evaluate its lead candidate MS1819-SD as a treatment for patients with exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF). This announcement follows the approval by the U.S. Food and…
The Cystic Fibrosis Foundation (CFF) will spend $100 million over the next five years to fund research aimed at fighting infections in patients with cystic fibrosis (CF) — a problem that seems destined to grow as patients live longer with the disease. The announcement came during the 32nd…
Two compounds that make up approved cystic fibrosis (CF) therapy Orkambi — lumacaftor and ivacaftor — may work more effectively in patients whose airways are inflamed, according to new research. By mimicking the inflamed CF airways of patients in the lab, researchers also established a new…
A therapy that involves a synthetic cationic antimicrobial peptide (CAP) can decrease Pseudomonas aeruginosa biofilm formation alone or in combination with another antimicrobial agent called tobramycin. Cystic fibrosis (CF) patients are susceptible to repeated bacterial infections. Infection with Pseudomonas aeruginosa (P. aeruginosa) is particularly harmful, since it has been shown to decrease…
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