Rare Disease Report

American Society of Clinical Oncology 2018

Pauline Funchain, MD, discusses the use of multiplex germline testing for rare diseases and cancers and explains the benefits of uncovering genetic factors.
Owen A. O’Connor, MD, PhD, outlines the importance of clinical trials and reviewed strides made in lymphoma treatments. 
Dr Meletios A. Dimopoulos, discusses the use of ibrutinib combined with rituximab for the treatment of patients with Waldenström macroglobulinemia.
The 2018 American Society of Clinical Oncology (ASCO) Annual Meeting held in Chicago, Illinois, June 1-5, 2018, featured data from numerous studies and advancements being made in rare cancers. Here are 5 key takeaways from the meeting.  
Stay up-to-date on the latest rare disease news by reading the top 5 articles of the week.
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).
The use of the maintenance regimen increased both 5-year disease-free survival (DFS) and 5-year overall survival (OS) in rhabdomyosarcoma.
 
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.
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