Rare Disease Report
Patients & Caregivers


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
The company is optimistic it can meet the requirements asked by the FDA without the need for more studies.
Vertex announced that the FDA and EMA have accepted applications for tezacaftor/ivacaftor combination treatment in a subset of patients with cystic fibrosis.
Regeneron's Suptavumab (REGN2222) in respiratory syncytial virus did not meet its endpoint, and the company will discontinue development of the drug.
The FDA granted orphan drug designation to tesevatinib for treatment of non-small cell lung cancer with epithelial growth factor receptor-activating mutations.
Aradigm has submitted its NDA for Linhaliq for the treatment of non-cystic fibrosis bronchiectasis (NCFBE) due to P. aeruginosa.
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