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March 15, 2019March 15, 2019

Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn

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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 ... Read more

March 14, 2019March 14, 2019

New Antibiotic Formulation Better at Controlling Infections in Bronchiectasis

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Inhaled antibiotics have been proposed as efficient treatments to reduce bacterial infections in bronchiectasis patients. Now researchers have shown that a new formulation of antibiotics using nanoparticles, called nanoplex, ... Read more

March 11, 2019March 11, 2019

Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist

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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 ... Read more

March 7, 2019March 7, 2019

Case Report Illustrates Risk of Bronchiectasis in People Not Vaccinated Against Measles

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The case of a man whose measles infection as a child was linked to his later development of bronchiectasis draws attention to the risk of the bronchiectasis due to fewer vaccinations ... Read more

March 6, 2019March 6, 2019

NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research

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Rare diseases affect about 30 million Americans — roughly the same number as those with type 2 diabetes. Yet only 5 percent of the estimated 7,000 rare diseases known ... Read more

February 28, 2019February 28, 2019

FDA Maintains Refusal of Aradigm’s Bronchiectasis Bacteria Therapy and Recommends Further Study

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The U.S. Food and Drug Administration (FDA) has stood by its initial refusal to approve Apulmiq for the treatment of non-cystic fibrosis bronchiectasis (NCFBE) associated with chronic Pseudomonas aeruginosa lung infection. The agency ... Read more

February 25, 2019February 25, 2019

WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April

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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 ... Read more

February 21, 2019February 21, 2019

Lower Oxygen Levels During Exercise Is a Risk Factor for Osteoporosis in Non-CF Bronchiectasis, Study Reports

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Decreased blood oxygen levels during the six-minute walk test (6MWT) is a risk factor for osteoporosis in patients with non-cystic fibrosis (CF) bronchiectasis, according to a study. The findings ... Read more

February 20, 2019February 20, 2019

Vienna to Host RARE2019 Meeting on Rare Diseases

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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 ... Read more

February 14, 2019February 14, 2019

Glittre Test Can Help Evaluate Functional Capacity and Activities of Daily Living in Bronchiectasis Patients

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The Glittre activities of daily living (ADL) test can help evaluate functional capacity and daily living activities in patients with bronchiectasis, a new study shows. The study, “Cardiorespiratory Responses to ... Read more

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