Rare Disease Report


The FDA has approved pembrolizumab for the treatment of refractory primary mediastinal large B-cell lymphoma (PMBCL) or who have relapsed after 2 or more previous lines of therapy.
The first patient has been dosed in Actinium Pharmaceuticals’ phase 1 trial evaluating Actimab-A in combination with CLAG-M for patients with relapsed or refractory (r/r) acute myeloid leukemia (AML).
Ruben Mesa, MD, director at Mays Cancer Center at UT Health San Antonio and MD Anderson Cancer Center, discusses myeloproliferative neoplasms and other rare cancers.
Two single-patient, Compassionate Use trials for RT001 have been initiated for late onset Tay Sachs (LOTS) disease and familial encephalopathy with neuroserpin inclusion bodies (FEIN or neuroserpinosis). 
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.
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