Rare Disease Report
Patients & Caregivers


Aradigm Corporation’s meeting with the Antimicrobial Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) is slated for January 11, 2018.
Additional clinical practice guidelines were published as it pertains to to diagnosing lymphangioleiomyomatosis and the management of pneumothoraces in patients with it.
$750,000 research grant program for clinical scientists to establish CF research programs
At the ACAAI Meeting, Dr Timothy Craig presented data showing the prophylactic use of Haegarda reduced attacks by 98% in severe HAE patients
Today, Boehringer Ingelheim previewed the data to be presented at the 2017 CHEST Annual Meeting in Toronto, Canada next week.
Pulmatrix's Pulmazole (PUR1900), designed to fight pulmonary fungal infections, has been designated as a Qualified Infectious Disease Product by the FDA.
PBS’s documentary The Gene Doctors is available. Film explores the scientific breakthroughs that are transforming rare disease patients
As CF patients are living longer, their hospital costs are also increasing. Costs were $500 million in 2003 vs $1.19 billion in 2013.
Aradigm announced the FDA acceptance of its NDA for Linhaliq to treat patients with non-cystic fibrosis bronchiectasis with chronic infections from P. aeruginosa.
Administration of CSL830 twice weekly at doses 40 IU per kilogram or 60 IU per kilogram has a significant preventive effect
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