Challenges
Experts share their insights in diagnosing and managing rare conditions
Future Treatment Options for Multiple Myeloma
Amrita Krishnan, MD, hematologist-oncologist at the City of Hope Comprehensive Cancer Center talks about what the future holds for managing people with multiple myeloma.
FDA Approves Drug to Treat Gastrointestinal Stromal Tumor (GIST) Based on Phase I Study
The U.S. Food and Drug Administration (FDA) has approved Ayvakit (avapritinib) for treating adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) with a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation.
CME Program for Eosinophilic Granulomatosis with Polyangiitis (EGPA)
Michael E. Wechsler, MD, MMSc, Professor of Medicine at National Jewish Health and University of Colorado School of Medicine talks about the need for clinicians to be more aware about Eosinophilic granulomatosis with polyangiitis (EGPA).
Why Joint Pain Is Common in Hemophilia
Gallia Levy, MD, PhD, Associate Group Medical Director, at Genentech explains why joint pain is so prevalent in persons with Hemophilia A.
Programmed Cell Death Following Hemorrhagic Stroke
Rajiv Ratan, MD, PhD, of the Burke Neurological Institute, explains the mechanisms involved in how cells undergo ‘programmed death’ following a hemorrhagic stroke.
Surgery for Thyroid Eye Disease
Gary Joseph Lelli, MD, of the Department of Ophthalmology at Weill Cornell Medicine discusses surgical options currently available to treat thyroid eye disease due to Graves’ disease.
FDA Approves Vaccine to Prevent Ebola Virus Disease
The U.S. Food and Drug Administration (FDA) has approved the first vaccine to prevent Ebola virus disease. The vaccine, Ervedo, by Merck & Co, Inc, was granted approval based on a study conducted during the 2014-2016 outbreak in Guinea. The study involved 3,537...
FDA Committee Votes in Favor or New Epithelioid Sarcoma Treatment
The Oncologic Drugs Advisory Committee (ODAC) of the U.S. Food and Drug Administration (FDA) voted unanimously (11 – 0) in favor of approving tazemetostat to treat patients with epithelioid sarcoma.
Oxbryta (Voxelotor) to Treat Sickle Cell Disease
Jonathan Sorof, MD, of Global Blood Therapeutics (GBT) explains the mechanism of action of Oxbryta (voxelotor) to treat persons with sickle cell disease.
FDA Approves Second Exon-Skipping Drug for Duchenne
The U.S. Food and Drug Administration has approved Vyondys 53 (golodirsen) to treat patients with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. Golodirsen is an antisense oligonucleotide that can ‘skip’ over aspects of the RNA to make person’s with...