Rare Disease Report
Patients & Caregivers


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.
CSL Behring announced Haegarda is now available for routine prophylaxis use to prevent hereditary angioedema (HAE) attacks.
In the Lancet, Dr Marc Riedl et al have published results of the Phase 2 study assessing Ruconest for the prevention of HAE attacks.
IPF is a chronic, progressive fibrotic interstitial pneumonia that does not have a clear cause.
On Tuesday, Vertex Pharmaceuticals announced positive data from Phase 1 and Phase 2 studies of three different triple combination regimens in people with cystic fibrosis (CF) who have one F508del mutation and one minimal function mutation (F508del/Min).
Most people do not become ill from Nontuberculous mycobacteria (NTM), but in people of compromised health, an infection can lead to more serious problems.
Interim data from an ongoing, open-label, pilot Phase 2 study of XmAb5871 in patients with IgG4-Related Disease (IgG4-RD) was presented today at EULAR 2017.
Nucala (mepolizumab) helped about one-half of the patients with refractory or relapsed eosinophilic granulomatosis with polyangiitis a patient population that has been historically difficult to treat.
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