Rare Disease Report


hATTR compass program will empower people with accurate genetic information so they can make informed decisions about their healthcare.
Nicholas J Robert, MD, discusses treatment challenges associated with rare cancers like Merkel cell carcinoma.
Updated interim data from Affimed NV’s phase 1b dose-escalation study evaluating AFM13 for the treatment of relapsed/refractory Hodgkin Lymphoma were released at the 23rd Congress of the European Hematology Association.
Ray Comenzo, MD, highlights promising ANDROMEDA phase 3 trial, which evaluated the efficacy of subcutaneous daratumumab and cyclophosphamide, bortezomib, and dexamethasone (CyBorD) as a combination therapy for newly diagnosed light chain (AL) amyloidosis patients.
William D. Tap, MD, lead investigator of the ENLIVEN study provides latest update on pexidartinib as an effective treatment for patients with tenosynovial giant cell tumor (TGCT), especially the diffuse form.
The FDA has approved bevacizumab for patients with ovarian, fallopian tube, or primary peritoneal cancer in combination with carboplatin and paclitaxel, followed by single-agent bevacizumab, for either stage 3 or 4 disease after initial surgical resection.
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). 
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