Challenges

Experts share their insights in diagnosing and managing rare conditions
Approved and Emerging Therapies for Mucopolysaccharidoses (MPS)

Approved and Emerging Therapies for Mucopolysaccharidoses (MPS)

Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone Health, discusses some of the approved and emerging therapies for mucopolysaccharidoses (MPSs), a group of rare, inherited lysosomal storage disorders that are clinically characterized by abnormalities in multiple organ systems and reduced life expectancy.

Typical Physical Features Across All SubTypes of Mucopolysaccharidoses (MPSs)

Typical Physical Features Across All SubTypes of Mucopolysaccharidoses (MPSs)

Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone Health, discusses some of the typical physician features across all subtypes of ,mucopolysaccharidoses (MPSs), a group of rare, inherited lysosomal storage disorders that are clinically characterized by abnormalities in multiple organ systems and reduced life expectancy.

2030 Goal: Universal Health Coverage for Rare Diseases

2030 Goal: Universal Health Coverage for Rare Diseases

Rare Disease International (RDI) is a global alliance of rare disease patient organizations designed to be an international voice for the rare disease community. One of its key initiatives is to include rare diseases in the Universal Health Coverage being developed by the United Nations (UN) for the year 2030.

Mucopolysaccharidosis type IV (MPS IV or Morquio Disease)

Mucopolysaccharidosis type IV (MPS IV or Morquio Disease)

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 IV (MPS IV) or Morquio disease.

Fibromuscular Dysplasia and the “String of Beads”

Fibromuscular Dysplasia and the “String of Beads”

Jeffrey W Olin, DO, Professor of Medicine at the Icahn School of Medicine at Mount Sinai, discusses the “string of beads” as the description typically given to the appearance of the renal artery in fibromuscular dysplasia (FMD).

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