Hunter Syndrome (MPS Type II) Overview

  Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City discusses Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome. This condition affects many different parts of the body and occurs almost...

ELX-02: Emerging Therapy for Cystic Fibrosis

  Mr. Robert (Bob) E. Ward, Chairman of the Board and Chief Executive Officer of Eloxx Pharmaceuticals provides an overview of his company’s leading drug candidate, ELX-02 for cystic fibrosis. ELX-02 is designed to increase the read-through activity in...

Pulmonary Arterial Hypertension (PAH): Emerging Treatment Options

  Roxana Sulica, MD, Associate Professor, Department of Medicine and Director, Pulmonary Hypertension at NYU Langone in New York City discusses some of the emerging tretment options for pulmonary arterial hypertension (PAH). One of these emerging therapies is the...

Challenges of Diagnosing Patients with 22q11.2 Deletion Syndrome

Bernice Morrow, PhD, Director of Translational Genetics, Albert Einstein College of Medicine, discusses some of the challenges of diagnosing patients with 22q11.2 deletion syndrome, a disorder caused by the deletion of a small piece of chromosome 22. 22q11.2 deletion...
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