Lysosomal Storage Disorders

Apr 19, 2021| Posted in: Lysosomal Storage Disorders, Treatment

New CME Program Focused on the Latest Gaucher Disease Research

Professor Derralynn Hughes, MD of the Royal Free…
Apr 19, 2021| Posted in: Lysosomal Storage Disorders, Treatment

Live CME Webinar: Current and Emerging Lysosomal Storage Disease Therapies

This Tuesday, April 20th, join Drs Ozlem Goker-Alpan…
Mar 18, 2021| Posted in: Lysosomal Storage Disorders, Regulations, Treatment

Why is Newborn Screening So Confusing?

Damara Ortiz, MD, FAAP, FACMG, Assistant Professor of…
Mar 17, 2021| Posted in: Lysosomal Storage Disorders, Metabolic Disorders, Neurology/Nervous System Diseases

What is Metachromatic Leukodystrophy?

Francesca Fumagalli, PhD, Researcher of Cardiovascular Medicine at…
Mar 12, 2021| Posted in: Heart Diseases, Lysosomal Storage Disorders, Neurology/Nervous System Diseases

New ERT for Pompe Disease As Effective as Older ERT

Priya Kishnani, MD, Professor of Pediatrics at the…

Gaucher Disease Research Highlights

This CME activity highlights the latest information about Gaucher disease and provides expert analysis of its clinical relevance.

Current and Emerging Therapies for Lysosomal Storage Disease

Drs. Goker-Alpan and Zimran provide an overview of current and emerging therapies for lysosomal storage diseases in this CME program.

Niemann–Pick Disease Type C Learning Center

Niemann-Pick disease type C (NPC) is a disabling, lysosomal storage disorder that has been diagnosed prenatally, neonatally, during childhood, and even into adulthood.

Fabry Disease Research Highlights

This CME activity highlights the latest information about Fabry disease from WORLDSymposium 2021.

Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency Learning Center

Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare, genetic disorder that affects the central nervous systems of young patients.

Mucopolysaccharidosis I CME Program

Paul Orchard, MD from the University of Minnesota Medical School provides an overview of Mucopolysaccharidosis I in these CME and CNE courses. Dr. Orchard discusses the importance of newborn screening and genetic counseling, and the challenges of diagnosing and treating patients with MPS I.

Lysosomal Storage Disorders

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