by Peter Ciszewski | Jun 7, 2019
The process for researching and developing new medicines is notoriously time-consuming, complicated and costly – and even more so when it comes to therapies for rare diseases. Shire is now Takeda. David Whiteman, MD, Global Development Team Lead, Rare Metabolic...
by Peter Ciszewski | Jun 7, 2019
Ido Weinberg, MD, Medical Director, VASCORE; Assistant Professor of Medicine Harvard Medical School, Co-Medical Director, Anticoagulation Management Services, Massachusetts General Hospital, discusses Fibromuscular Dysplasia (FMD) in men and women and its...
by Peter Ciszewski | Jun 6, 2019
Debra Miller, founder of CureDuchenne, discusses research activity in Duchenne muscular dystrophy (DMD) and the role of the advocate in this rare disease. Debra founded CureDuchenne in 2003 with her husband after their only son was diagnosed with Duchenne. The...
by Peter Ciszewski | Jun 5, 2019
Heather A. Lau, MD, Assistant Professor, Department of Neurology; Associate Director, Division of Neurogenetics; Director, Lysosomal Storage Disease Program at NYU Langone Health provides an overview of Mucopolysaccharidosis type VI (MPS VI). MPS VI, also known...
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...
