Rare Disease Report

Oncology

Robert Chen, MD, and Hodgkin’s lymphoma patient, Jeremy Hernandez, speak on the challenges of receiving and treating a rare cancer diagnosis in young adults.
 
A team of investigators has found that collaborative efforts among several leading medical institutions have helped increase the study of new medications for myelodysplastic syndromes (MDS).
Interferon‐α2 and ruxolitinib combination therapy shows promise in treating myeloproliferative neoplasms, according to recent preliminary findings.
Karlyne Reilly, PhD, discusses the Rare Tumor Patient Engagement Network and the importance of getting patients with rare conditions involved in cancer research.
The FDA has granted a priority review to combination therapy, ibrutinib/obinutuzumab, for the treatment of previously untreated adult patients with chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL).
 
John Hopper, MBA, highlights advances made in the fight against rare cancers and discusses what’s next for the community.
Although there have been many successes experienced in the oncology community, more genomic data is needed.
 
The FDA has granted an orphan drug designation to MediciNova Inc.’s MN-166, an adjunctive therapy to temozolomide treatment for patients with glioblastoma.
Stay up-to-date on the latest rare disease news by reading the top 5 articles of the week.
Robert J. Kreitman, MD, discusses the FDA’s recent approval of moxetumomab pasudotox (Lumoxiti) for the treatment of hairy cell leukemia (HCL).
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