Rare Disease Report
Patients & Caregivers


Arrowhead Pharmaceuticals Inc. announced that the company’s second-generation investigational medicine ARO-AAT was granted orphan drug designation by the FDA.
Faron Pharmaceuticals announced this morning that the U.S. FDA has approved the Investigational New Drug application for Traumakine (FP-1201-lyo) for the treatment of Acute Respiratory Distress Syndrome.
Rare Disease Report spoke with pulmonologist Dr. Marlies S. Wijsenbeek, Department of Respiratory Medicine, Erasmus University Medical Center (Rotterdam The Netherlands) about newly-published data in IPF.
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
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