Larry Luxner,  —

Hill-Rom, which produces the Monarch Airway Clearance System for patients with cystic fibrosis (CF) and other diseases, has put its product on a “temporary shipping hold” as it scrambles to affix labels to 60 of the devices. According to a Class 2 recall notice posted by…

Jim Best, a 34-year-old Canadian pharmacist with cystic fibrosis, is also a talented photographer who rides his motorcycle across the country every year to raise funds for CF research. In addition to generating funds, the trips help the Sackville, Nova Scotia, resident spread awareness about the incurable disease that affects…

The National Organization for Rare Disorders (NORD) says it’s “disappointed and dismayed” after the House of Representatives voted 227-205 last week to repeal the Orphan Drug Tax Credit as part of a U.S. tax reform package. A similar package before the Senate Finance Committee does not repeal the credit…

More adults than children are living with cystic fibrosis for the first time ever, says the Cystic Fibrosis Foundation (CFF). This means more patients are encountering issues they have never had to face — such as saving for their children’s college and their own retirement. They are also having to deal…

Children’s National Health System no longer treats just kids. Its Rare Disease Institute, launched in April 2017, has partnered with the National Organization for Rare Disorders (NORD) to become the first of many U.S. “centers of excellence” to look after patients with rare diseases, regardless of age. The effort…

About 4,400 researchers, doctors, patients and others from 46 countries are converging on Indianapolis for the 31st Annual North American Cystic Fibrosis Conference — the world’s largest gathering dedicated to cystic fibrosis research and care. The Nov. 2-4 event at the Indiana Convention Center will feature more than 350 sessions and…

More therapies are now available for the 30 million or so people with rare diseases in the U.S. than ever before, and millions of dollars are being invested in clinical studies that will test new ways of evaluating — and advancing — potential treatments, including the use of natural history…

As Congress begins debate this week to overhaul the U.S. tax code, lawmakers should leave the Orphan Drug Act (ODA) — and the tax incentives it offers pharmaceutical companies to develop therapies for rare diseases — off the table. That’s the message being pushed by the National Organization for…

A cystic fibrosis expert will be among more than 80 speakers at this month’s Rare Disease & Orphan Products Breakthrough Summit in Washington. The nonprofit National Organization for Rare Disorders (NORD) will be hosting the sixth annual event Oct. 16-17 at the Marriott Wardman Park Hotel. It is expected to attract…

The U.S. Food and Drug Administration (FDA) has approved Kalydeco (ivacaftor) to treat more than 600 of the estimated 30,000 Americans with cystic fibrosis (CF), Vertex Pharmaceuticals announced. The oral medicine may now be prescribed to CF patients ages 2 and older who have one of five residual…