John Maraganore Speaks Out on His Role as Bio Chair

Dr. John Maraganore has served as the CEO and a Director of Alnylam since 2002 and is the Chair of the Biotechnology Innovation Organization (BIO) for the 2017-2018 term. Dr. John Maraganore has led a career pursuing therapies to address unmet medical needs. At...

Overview of Sly Syndrome (Mucopolysaccharidosis type VII)

  Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City discusses Mucopolysaccharidosis type VII (MPS VII), also known as Sly syndrome. MPS VII is a progressive condition that affects most tissues and organs. The severity...

FDA Approves Palynziq for PKU, a Rare Genetic Disease

The U.S. Food and Drug Administration today approved Palynziq (pegvaliase-pqpz) for adults with a rare and serious genetic disease known as phenylketonuria (PKU). Patients with PKU are born with an inability to break down phenylalanine (Phe), an amino acid present in...

Overview of Lysosomal Storage Disorders

  Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City discusses lysosomal storage diseases, a group of inherited metabolic diseases that are characterized by an abnormal build-up of various toxic materials in the...

Epidermolysis Bullosa Overview

  Daniel de Boer, Founder and CEO of ProQR, discussuses Epidermolysis bullosa (EB), a group of genetic skin diseases that cause the skin to blister and erode very easily. In people with EB, blisters form in response to minor injuries or friction, such as rubbing...
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