Author Archives: Iqra Mumal MSc

When it comes to lung transplants, a study has found that the practice of many cystic fibrosis (CF) specialists differs from that of updated recommendations, highlighting a need to educate providers on new guidelines. The study, “Lung transplant referral practice patterns: a survey of cystic fibrosis physicians and general…

Kristine Durkin, a West Virginia University graduate student, is carrying out a study to assess whether teenagers with cystic fibrosis (CF) are meeting their nutritional requirements, and identify ways to help them improve their diets. CF teenagers often struggle to maintain healthy weight. They usually receive two to three…

Canadian patient advocates and leaders in the field of cystic fibrosis (CF) are meeting with Canadian government officials to lobby for changes to proposed regulatory reforms that are preventing CF patients from accessing Vertex’s new breakthrough therapy, Trikafta. Trikafta, Vertex’s most recent FDA-approved medication for the…

Vertex Pharmaceuticals said the worldwide COVID-19 outbreak has not affected its supply chain or its 2020 business outlook. The company said it’s confident it can continue to supply all its approved medications to patients with cystic fibrosis (CF) around the world. COVID-19 — an acronym for COrona…

Eloxx Pharmaceuticals anticipates reporting topline data from the Phase 2 clinical trial program for ELX-02 — its lead therapeutic candidate to treat cystic fibrosis caused by nonsense mutations — during the first half of 2020, the company has announced. “We are committed to ensuring full enrollment of our Phase…

The National Institute for Health and Care Excellence (NICE) in the U.K. is currently in the process of evaluating Vertex Pharmaceuticals’ triple combination therapy — known as Trikafta in the United States — for the treatment of cystic fibrosis (CF). The institute expects to deliver its…

The U.S. Food and Drug Administration (FDA) has granted its orphan drug designation to ARV-1801 (sodium fusidate tablets), a treatment developed by Arrevus for patients with cystic fibrosis (CF). CF patients frequently develop lung infections due to the accumulation of thick mucus in their lungs, creating…

The Cystic Fibrosis Foundation has reached an agreement with Synspira Therapeutics to support the further development of SNSP003, an enzyme replacement therapy (ERT) designed to treat malabsorption syndromes, including those affecting patients with cystic fibrosis (CF). Malabsorption syndromes are a group of disorders that develop when there…

Allergan has entered into a definite agreement to sell Zenpep (pancrelipase), a therapy for exocrine pancreatic insufficiency due to cystic fibrosis (CF) and other conditions. The agreement was reached alongside the currently ongoing regulatory approval process for AbbVie’s acquisition of Allergan, and is contingent upon approval of…

The Cystic Fibrosis Trust is expanding its network of cystic fibrosis (CF) centers in the United Kingdom (U.K.) to give more patients access to clinical trials. Nine CF centers were chosen to be part of the Trust’s Clinical Trials Accelerator Platform based on their interest in clinical trials…