News

February 20, 2018February 20, 2018

Patient Advocacy Groups Worldwide Plan Events to Mark Rare Disease Day, Feb. 28

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A rock-painting contest in Las Vegas. A fashion show in New York. A 7,000-meter race around the Washington Monument that’ll coincide with a similar #Racefor7 event in Bengaluru and Mumbai, ... Read more

February 15, 2018February 15, 2018

Stem Cell Transplants Rejuvenate Chinese Bronchiectasis Patients’ Damaged Lungs

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Chinese medical scientists used stem cell transplants to rejuvenate the damaged lungs of two bronchiectasis patients, a study reports. An article on the pilot clinical trial’s results appeared in the journal ... Read more

February 12, 2018March 15, 2018

Rare Disease Groups, Patients Differ on ‘Right to Try’ Bill Before US Congress

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David Curtis Glebe, a retired 64-year-old public prosecutor now living in Millsboro, Delaware, knows he’s lucky to be alive. In mid-2013, while in Arizona, Glebe was diagnosed with pancreatic neuroendocrine ... Read more

February 8, 2018February 8, 2018

S. aureus Infection Not Strongly Linked to Decline in Bronchiectasis Patients, Study Reports

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An airway infection caused by Staphylococcus aureus is not an independent risk factor for more severe disease in bronchiectasis patients, a study published in the Annals of the American Thoracic ... Read more

January 31, 2018January 30, 2018

NORD Fundraiser Sets 7,000-Mile ‘Bike, Run or Walk’ Goal for 7,000 Rare Diseases

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This week marks the launch of the “7,000 Mile Rare Movement,” a nationwide effort to raise money for research into the 7,000 known rare diseases that afflict at least 30 ... Read more

January 30, 2018January 30, 2018

Aradigm’s Linhaliq NDA for Non-CF Bronchiectasis Fails to Get FDA Approval

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The U.S. Food and Drug Administration (FDA) told Aradigm the agency could not approve the company’s New Drug Application (NDA) for Linhaliq in its present form. Linhaliq is designed to treat patients with non-cystic ... Read more

January 23, 2018January 23, 2018

An Antibiotic-resistant Bacteria Worsens Bronchiectasis, but Does Not Increase Death Rates, Study Shows

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An antibiotic-resistant Pseudomonas aeruginosa infection worsens bronchiectasis, but does not increase patients’ death rates, a Chinese study shows. The research, “Antibiotic-resistant Pseudomonas aeruginosa infection in patients with bronchiectasis: prevalence, risk factors and prognostic implications,” ... Read more

January 16, 2018January 16, 2018

FDA Advisory Committee Not Convinced of Linhaliq’s Benefits in Bronchiectasis Infections

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An advisory committee is recommending against U.S. regulatory approval of a mist formulation that Aradigm developed to deliver an antibiotic for the Pseudomonas aeruginosa lung infections in bronchiectasis. Although the U.S. Food and Drug ... Read more

January 9, 2018January 9, 2018

Bronchiectasis May Trigger Autoimmune Inflammatory Disease, Study Suggests

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Bronchiectasis is commonly found in patients with autoimmune conditions, causing blood vessel inflammation, a recent study found. Moreover, the presence of bronchiectasis in these patients is linked to specific disease ... Read more

January 4, 2018January 4, 2018