Lysosomal Storage Disorders

May 27, 2020| Posted in: Congenital And Genetic Conditions, Drug Development, Lysosomal Storage Disorders

Protalix’ Fabry Disease Clinical Program

Dror Bashan, President and CEO of Protalix Biotherapeutics…

May 15, 2020| Posted in: Congenital And Genetic Conditions, Heart Diseases, Hematologic Disorders, Kidney And Urinary Diseases, Lysosomal Storage Disorders, Metabolic Disorders, Neurology/Nervous System Diseases, Ophthalmology/Eye Diseases, Skin Conditions

Fabry Disease Research Highlights: Earn CME/CNE Credit

Ozlem Goker-Alpan, MD, provides a summary of research…

May 9, 2020| Posted in: Advocacy, Congenital And Genetic Conditions, Diagnosis, Expert Perspectives, Heart Diseases, Lysosomal Storage Disorders, Metabolic Disorders, Neurology/Nervous System Diseases, Treatment

Podcast: Diagnosing Gaucher Disease Before the ERT Era

Cyndi Frank, of the Gaucher Community Alliance explains…

May 7, 2020| Posted in: Cancers, Diagnosis, Hematologic Disorders, Lysosomal Storage Disorders, Metabolic Disorders, News, Treatment

Journal Watch: COVID-19, Neimann-Pick, Sickle Cell, Multiple Myeloma

CheckRare Journal Watch: COVID-19, Neimann-Pick, Sickle Cell, Multiple…

Apr 30, 2020| Posted in: Congenital And Genetic Conditions, Expert Perspectives, Heart Diseases, Kidney And Urinary Diseases, Lysosomal Storage Disorders, Metabolic Disorders, Neurology/Nervous System Diseases, Ophthalmology/Eye Diseases, Skin Conditions

Fabry Disease Overview

Ozlem Goker-Alpan, MD, founder of the Lysosomal &…

Apr 29, 2020| Posted in: Congenital And Genetic Conditions, Drug Development, Expert Perspectives, Heart Diseases, Hematologic Disorders, Lysosomal Storage Disorders, Neurology/Nervous System Diseases

Gene Therapies in Development for Gaucher Disease

Neil Weinreb, MD, FACP provides a summary of…

Apr 23, 2020| Posted in: Drug Development, Expert Perspectives, Hematologic Disorders, Lung Diseases, Lysosomal Storage Disorders, Neurology/Nervous System Diseases, Treatment

Enzyme Replacement Therapy Versus Gene Therapy

Dwight Koeberl, MD, PhD of Duke University School…

Apr 17, 2020| Posted in: Drug Development, Expert Perspectives, Lung Diseases, Lysosomal Storage Disorders, Metabolic Disorders, News, Ophthalmology/Eye Diseases, Treatment

The Latest Developments in Pompe Disease Research

Priya Kishnani, MD of Duke University School of…

Apr 14, 2020| Posted in: Autoimmune / Autoinflammatory Disorders, Congenital And Genetic Conditions, Expert Perspectives, Lung Diseases, Lysosomal Storage Disorders, Neurology/Nervous System Diseases, News

Webinar: COVID-19 and Fabry Disease

Heather Lau, MD, Director of the Lysosomal…

Apr 13, 2020| Posted in: Autoimmune / Autoinflammatory Disorders, Congenital And Genetic Conditions, Expert Perspectives, Lysosomal Storage Disorders, Neurology/Nervous System Diseases, News, Ophthalmology/Eye Diseases

Webinar: COVID-19 and Pompe Disease

Heather Lau, MD, Director, Lysosomal Storage Disease Program…

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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