by Peter Ciszewski | Jun 4, 2019
Jeffrey W Olin, DO, Professor of Medicine at the Icahn School of Medicine at Mount Sinai, discusses diagnosing and treating fibromuscular dysplasia (FMD), a rare condition characterized by abnormal development or growth of cells in the walls of blood vessels...
by Peter Ciszewski | Jun 3, 2019
Ido Weinberg, MD, Medical Director, VASCORE; Assistant Professor of Medicine, Harvard Medical School, Co-Medical Director, Anticoagulation Management Services, Massachusetts General Hospital, discusses the importance of raising awareness of fibromuscular...
by Peter Ciszewski | Jun 3, 2019
The U.S. Food and Drug Administration (FDA) granted Fast Track designation for PD-0325901, an investigational, oral, small molecule inhibitor of MEK1 and MEK2, for the treatment of patients 2 years of age or older with neurofibromatosis type 1-associated inoperable...
by Peter Ciszewski | Jun 3, 2019
Anne R. Pariser, MD, Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences at the National Institutes of Health discusses gene therapy and how the NIH is looking at “platform approaches” for treatments for...
by Peter Ciszewski | May 31, 2019
Peter Saltonstall, President and Chief Executive Officer of the National Organization for Rare Disorders (NORD), discusses his organization’s focus on the rare disease patient. For more than 30 years, NORD have been providing services for patients and...
