Lysosomal Storage Disorders
Jan 18, 2021| Posted in: Congenital And Genetic Conditions, Diagnosis, Lysosomal Storage Disorders, Metabolic Disorders, Neurology/Nervous System Diseases
Diagnosing Niemann-Pick Type C
Elizabeth Berry-Kravis, MD, PhD, co-director of the Molecular… Jan 15, 2021| Posted in: Congenital And Genetic Conditions, Diagnosis, Lysosomal Storage Disorders, Metabolic Disorders, Neurology/Nervous System Diseases
Overview of Niemann-Pick Type C
Elizabeth Berry-Kravis, MD, PhD, co-director of the Molecular… Jan 12, 2021| Posted in: Congenital And Genetic Conditions, Heart Diseases, Hematologic Disorders, Lysosomal Storage Disorders, Metabolic Disorders, Neurology/Nervous System Diseases, Ophthalmology/Eye Diseases, Skin Conditions, Treatment
Determining the Best Treatment Option for Fabry Disease
Robert Hopkin, MD, Associate Professor of Clinical Pediatrics… Jan 8, 2021| 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
Overview of Fabry Disease
Robert Hopkin, MD, Associate Professor of Clinical Pediatrics… Jan 4, 2021| Posted in: Autoimmune / Autoinflammatory Disorders, Cancers, Congenital And Genetic Conditions, Drug Development, Endocrine Disorders, Gastrointestinal Diseases, Heart Diseases, Hematologic Disorders, Kidney And Urinary Diseases, Lung Diseases, Lysosomal Storage Disorders, Metabolic Disorders, Musculoskeletal Diseases, Neurology/Nervous System Diseases, News, Ophthalmology/Eye Diseases, Skin Conditions, Treatment
Orphan Drugs Currently Under Review by the FDA
Every potential Food and Drug Administration (FDA) approval… Oct 29, 2020| Posted in: Congenital And Genetic Conditions, Lung Diseases, Lysosomal Storage Disorders, Metabolic Disorders, Neurology/Nervous System Diseases, Ophthalmology/Eye Diseases
New Treatments May Be Available for Niemann-Pick Disease Soon
Joslyn Crowe, executive director of the National Niemann-Pick… Oct 17, 2020| Posted in: Advocacy, Autoimmune / Autoinflammatory Disorders, Congenital And Genetic Conditions, Lysosomal Storage Disorders, Metabolic Disorders, Neurology/Nervous System Diseases, News
How Families Cope with a New Rare Disease Diagnosis
Joslyn Crowe, executive director of the National Niemann-Pick… Oct 2, 2020| Posted in: Advocacy, Autoimmune / Autoinflammatory Disorders, Cancers, Congenital And Genetic Conditions, Diagnosis, Drug Development, Endocrine Disorders, Featured, Gastrointestinal Diseases, Heart Diseases, Hematologic Disorders, Kidney And Urinary Diseases, Lung Diseases, Lysosomal Storage Disorders, Metabolic Disorders, Musculoskeletal Diseases, Neurology/Nervous System Diseases, News, Ophthalmology/Eye Diseases, Regulations, Skin Conditions, Treatment
A Preview of NORD’s Rare Summit 2020
Peter Saltonstall, President and Chief Executive Officer of… Sep 29, 2020| Posted in: Autoimmune / Autoinflammatory Disorders, Congenital And Genetic Conditions, Lysosomal Storage Disorders, Neurology/Nervous System Diseases, Treatment
Influence of COVID-19 on Lysosomal Storage Disease Patients in Israel
Shoshana Revel-Vilk, MD, Shaare Zedek Medical Center, Israel… Sep 24, 2020| Posted in: Expert Perspectives, Lysosomal Storage Disorders