Mucopolysaccharidosis type VI (MPS VI or Maroteaux-Lamy Syndrome)

  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...

Diagnosing and Treating Fibromuscular Dysplasia (FMD)

  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...

Raising Awareness of Fibromuscular Dysplasia (FMD)

  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...

Gene Therapy and Platform Approaches to Treating Rare Diseases

  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...
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