Rare Disease Report


E. Anders Kolb, MD, discusses the exciting possibilities of using genomic technologies to stratify pediatric cancer patients.
The FDA has granted a priority review for an anti-PD-1 therapy for the treatment of adults and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma.
Stay up-to-date on the latest rare disease news by checking out our top 5 articles of the week.
Investigators from McGill University have discovered a new link connecting immune cells and the development of gastric tumors in those with Peutz-Jeghers Syndrome.
The FDA has granted a fast track designation to CX-01 for the treatment of patients over 60 years of age receiving induction therapy for newly-diagnosed AML.
The FDA has approved ibrutinib (IMBRUVICA) plus rituximab (RITUXAN) for the treatment of adult patients with Waldenström's macroglobulinemia, a rare and incurable type of non-Hodgkin's lymphoma.
A rapid genetic diagnostic tool coupled with a sustained release therapeutic is being developed for lower-grade gliomas by a team of investigators.
The FDA has approved pembrolizumab to be used in combination with pemetrexed and platinum as first-line treatment for patients with metastatic, non-squamous non-small cell lung cancer, with no EGFR or ALK genomic tumor aberrations.
The FDA has granted an orphan drug designation to ASLAN Pharmaceuticals’ ASLAN003 for the treatment of acute myeloid leukemia.
The FDA has approved Bristol-Meyers Squibb Company’s nivolumab (Opdivo) for the treatment of metastatic small cell lung cancer (SCLC) that has progressed after platinum-based chemotherapy and at least 1 other line of therapy.
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