Rare Disease Report


Rigel announced that the U.S. FDA approved fostamatinib disodium hexahydrate (Tavalisse) tablets for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia.
Data presented at the 2018 American Association for Cancer Research Annual Meeting show that a new combination could provide long-term, systemic responses in patients with advanced melanoma.
The U.S. FDA granted Fast Track Designation for RP6530 (Tenalisib). Rhizen Pharmaceuticals S.A. is developing the drug for the treatment of patients with relapsed and/or refractory cutaneous T-cell lymphoma.
Results from a new study led by researchers in the CCR at the NCI could potentially explain why some patients with diffuse large B-cell lymphoma respond to treatment and others don’t.
Rare Disease Report tries to keep its audience at the forefront with the latest news from the hematological and oncological spaces. This new infographic provides 5 little-known facts about MPNs that might not necessarily be covered in news articles from RDR or other media outlets.
Rhizen Pharmaceuticals SA's product, Tenalisib (RP6530), is granted orphan drug designation from the FDA for the treatment of CTCL.
Molecular characteristics that link the genomic profiles of squamous cell carcinomas from 5 areas of the body have been uncovered by a team of researchers supported by the NIH.
Treatment with nivolumab, followed by ipilumab, produced significantly higher overall survival and objective response rates than sunitinib alone among intermediate- and poor-risk patients with previously untreated advanced renal-cell carcinoma.
The FDA has accepted BLA for hairy cell leukemia treatment.  
Phase 3 OLYMPUS clinical trial releases interim analysis of UGN-101 (MitoGel) for low-grade UTUC.
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