Rare Disease Report


The FDA has approved venetoclax for treatment of patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), with or without 17p deletion, who have received at least one prior therapy.
Planned analyses of phase 3 trial of gilteritinib as a maintenance therapy after allogeneic hematopoietic stem cell transplantation in patients with Fms-like tyrosine kinase 3 internal tandem duplication (FLT3-ITD) mutations in acute myeloid leukemia (AML). 
Updated research regarding the ANDROMEDA study indicates the efficacy of subcutaneous daratumumab (DARA SC) and cyclophosphamide, bortezomib, and dexamethasone (CyBorD) as a combination therapy in newly diagnosed light chain (AL) amyloidosis patients.
Study suggests a link between Lynch syndrome (LS) and more cancer types than previously thought.
The first patient has been dosed in the phase 2 expansion portion of its monotherapy trial of eFT508 for the treatment of relapsed, refractory non-germinal center B cell (non-GCB) diffuse large B cell lymphoma (DLBCL). 
Phase 1/2a clinical trial (NCT02253212) data on ultrasound-induced blood-brain barrier (BBB) opening for brain disorders like recurrent glioblastoma.
Iobenguane I 131 (AZEDRA) has been shown to be effective in patients with malignant, recurrent, or unresectable pheochromocytoma and paraganglioma (pheo/para).
Pauline Funchain MD, explains her research which focuses on assessing possible underlying genetic and familial factors in melanomas.
Radhakrishnan Ramchandren MD, highlights the recent US Food and Drug Administration’s (FDA) approval and advantages of ADCETRIS (brentuximab vedotin) in combination with chemotherapy for advanced stage, classic Hodgkin lymphoma.
Researchers present promising phase 2 data regarding tazemetostat for malignant mesothelioma, the first reported clinical data of an EZH2 inhibitor in these patients.
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