Rare Disease Report


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. 
Updated phase 1 data assessing indoximod plus front-line radiation and maintenance chemotherapy for the treatment of pediatric patients with DIPG proves promising.
Researchers from UT Southwestern Medical Center have identified 29 genes thought to contribute to rhabdomyosarcoma using the iExCN algorithm, a novel hybrid technology.
Teleconsulting technology through the European Network for Rare Adult Solid Cancer (EURACAN) will better help rare adult solid cancer patients achieve appropriate diagnoses and treatments.
Researchers find that JAK inhibitors used to treat myelofibrosis may actually lead to the development of aggressive lymphomas.
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