Neurology/Nervous System Diseases

Mar 30, 2021| Posted in: Congenital And Genetic Conditions, Musculoskeletal Diseases, Neurology/Nervous System Diseases

“I Call Myself Gadget Girl”: How One Patient Manages Her FSHD

Amy Bekier, facioscapulohumeral muscular dystrophy (FSHD) patient and…
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 12, 2021| Posted in: Advocacy, Endocrine Disorders, Neurology/Nervous System Diseases

More Research Needed To Understand Hypothalamic Obesity

Chelsea Johnson, mother of a boy with hypothalamic…

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.

Neurology/Nervous System Diseases

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