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