Lysosomal Storage Disorders

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…
Mar 8, 2021| Posted in: Advocacy, Congenital And Genetic Conditions, Lysosomal Storage Disorders, Regulations

Advice for Geneticists Performing Newborn Screenings

Damara Ortiz, MD, FAAP, FACMG, Assistant Professor of…

CME Program: Gaucher Disease Highlights from WORLDSymposium 2020

Neal Weinreb, MD, FACP highlights the latest information about Gaucher disease from WORLDSymposium and provides expert analysis of its clinical relevance to help health care providers better care for patients with this rare disease.

CME Program: Fabry Disease Highlights from WORLDSymposium 2020

Ozlem Goker-Alpan, MD highlights the latest Fabry disease information from the WORLDSymposium and provides expert analysis of its clinical relevance for members of the care team in order to help them care for patients with this rare disease.

CME Program: Mucopolysaccharidoses (MPSs) Highlights from WORLDSymposium 2020

Barbara K. Burton, MD highlights the latest information about MPSs from WORLDSymposium and provides expert analysis of its clinical relevance for members of the care team in order to help them care for patients with these rare diseases.

Lysosomal Storage Disorders

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