Rare Disease Report


Researchers find that blocking one or more genes in the HSF1 pathway could represent a new approach in treating the rare, aggressive disease.   
Investigators explore if polio can be used as a potential treatment for glioblastoma, an aggressive form of cancer that currently has no cure.
Yale Cancer Center scientists find that some rare hereditary cancer syndromes are defined by defects in homologous-recombination DNA repair.
Researchers map family trees of cancer cells in acute myeloid leukemia to better understand how the cancer responds to new drug, enasidenib.
Researchers say that adding a common altitude sickness drug to the treatment protocol for patients with glioblastoma may improve the effectiveness of chemotherapy and extend survival in some patients.
E. Anders Kolb, MD, outlines the challenges in diagnosing pediatric cancers and explains how molecular events define disease outcomes. 
Stay up-to-date on the latest rare disease news by reading the top 5 articles of the week.
The FDA has approved ivosidenib tablets for the treatment of adult patients with relapsed or refractory acute myeloid leukemia who have a specific genetic mutation.
The US Food and Drug Administration (FDA) has granted fast track designation to galinpepimut-S (GPS) for the treatment of multiple myeloma.
The FDA has granted a breakthrough therapy for atezolizumab Tin combination with bevacizumab for the treatment of advanced or metastatic hepatocellular carcinoma.
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