The Rare Disease Report page is a resource for medical news and expert insights on rare diseases. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for sickle cell disease, multiple myeloma, and more.
May 10th 2024
A Veterans Affairs study finds low alpha-1 antitrypsin phenotyping rates, indicating the need for improved healthcare provider education on AATD.
Physician and Patient Advocate Discuss Recent Drug Approval for Kidney Cancer
Saby George, MD, FACP, and Dena Battle, president of nonprofit patient advocacy group, KCCure, discussapproval of Opdivo (nivolumab) and Yervoy (ipilimumab) therpay combination for kidney cancer.
Genomic Link Discovered Between Rheumatoid Arthritis and Huntington
Upon unearthing rheumatoid arthritis’ epigenome, researchers at University of California San Diego School of Medicine and the Icahn School of Medicine at Mount Sinai have also discovered an overlap between the causes of rheumatoid arthritis and Huntington’s disease.
FDA Accelerates Eltrombopag Review for SAA Treatment
The FDA has accepted its supplemental New Drug Application (sNDA) and granted Priority Review designation to Promacta (eltrombopag) in combination with standard immunosuppressive therapy (IST) for first-line treatment of severe aplastic anemia (SAA).
FDA Grants Larotrectinib New Drug Application and Priority Review for Solid Tumors
FDA grants larotrectinib New Drug Application and Priority Review for the treatment of locally advanced or metastatic solid tumors containing an NTRK gene fusion on both adult and pediatric patients.