Larry Luxner,  —

When it comes to disease “awareness” days, weeks, and months, 2019 is crammed with hundreds of them. May leads the calendar with 37 such designations, calling attention to everything from stuttering and scleroderma to high blood pressure, lupus and skin cancer. Cystic Fibrosis Awareness Month also occurs in May. It’s…

Cumbersome security procedures, rising airfares, and shrinking legroom have made commercial air travel difficult enough these days — even for healthy passengers. Imagine how much harder it is for patients with rare diseases who must get to doctors’ appointments or clinical trials that are hundreds of miles away from home.

With an estimated 1.37 billion inhabitants, India will likely surpass China in five years as the world’s most populous country. That also means it will have more rare-disease patients than any nation. It already has more than twice as many as the 28-member European Union. Harsha K. Rajasimha, a genomics…

Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as scheduled, at the end of this month. Collin is an expert on the subject. For her University of Bath dissertation, she analyzed Brexit’s long-term impact…

With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases and the expensive therapies needed to treat them — particularly in an age of scarce economic resources — almost always entail “tragic choices,” warned Avraham Steinberg,…

The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., suburb. Some 1,200 people have already registered to attend the World Orphan Drug Congress (WODC) USA 2019, set to take place at the Gaylord National Harbor…

About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the 2nd International Congress on Advanced Treatments in Rare Diseases. The March 4-5 meeting, to take place at the Hilton Am Stadtpark Vienna, features 27 speakers on a variety of disorders…

Later this month, the Cystic Fibrosis Foundation (CFF) will host ResearchCon, its first virtual event dedicated to cystic fibrosis (CF) science and research. Some 480 people have registered for the Feb. 28 online conference, with at least 300 of them likely to participate, said Shellie Byrum, director of community…

Income level is the greatest single determinant of well-being in people with cystic fibrosis in the United States, said experts at a recent CF conference. Social worker Kim Reno, a manager at the Cystic Fibrosis Foundation’s Compass patient assistance program, estimates that actual healthcare determines only 20% of any…

Warning the reporter accompanying him not to take any pictures, veteran horticulturalist Michael Castleman punches an electronic code and unlocks the door to Room 209, nicknamed the “Mother Room.” Photography is indeed forbidden inside this living vault, which contains 20 phenotypes of cannabis plants thriving under the glare of 25…