Rare Disease Report


The FDA has granted a breakthrough therapy for atezolizumab Tin combination with bevacizumab for the treatment of advanced or metastatic hepatocellular carcinoma.
Researchers have identified a unique mutation in a deceased transthyretin (TTR) amyloidosis patient, which sheds light on the disease and may help identify new targets for treatment.
E. Anders Kolb, MD, emphasizes the difference between pediatric and adult cancers and the need to develop new treatments developed specifically for children.
Researchers at Baylor College of Medicine find that mutations in the gene TRAF7 are associated with a multisystem disorder presented by 7 patients.
Pauline Funchain, MD, discusses the use of multiplex germline testing for rare diseases and cancers and explains the benefits of uncovering genetic factors.
Study performed by researchers at Juntendo University reveals molecular mechanisms that trigger CALR-mutant myeloproliferative neoplasms (MPNs). 
AbbVie has submitted a supplemental new drug application to the FDA for venetoclax to be used in combination with HMA or LDAC for the treatment of AML patients who are ineligible for intensive chemotherapy.
Results from a phase 3 clinical trial evaluating an ibrutnib combination with a chemotherapy regimen for the treatment of diffuse large B-cell lymphoma fails to meet primary endpoint.
The FDA has accepted and granted priority review for a new supplemental Biologics License Application (sBLA) for pembrolizumab (KEYTRUDA) for the treatment of advanced hepatocellular carcinoma (HCC).
Owen A. O’Connor, MD, PhD, outlines the importance of clinical trials and reviewed strides made in lymphoma treatments. 
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