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rare disease

The ROS1ders advocacy group is 500 patients strong and they are helping researchers establish cell lines to improve understanding of rare ROS1-positve cancers. 

A New York Genome Consortium-led team plans retrospective and prospective analyses on very rare cancers in the hopes of improving treatment options available for patients.

Genome and RNA sequencing led to recurrent EGFR and BRAF rearrangements in cryptogenic congenital mesoblastic nephroma and infantile fibrosarcoma soft tumors.

This is the first US Food and Drug Administration approval for a drug based solely on data from a basket study.