Heart Diseases
Jan 18, 2021| Posted in: Congenital And Genetic Conditions, Drug Development, Heart Diseases, Treatment
Treatment Options for HoFH
James Underberg, MD, Clinical Assistant Professor of Medicine… 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… Dec 4, 2020| Posted in: Congenital And Genetic Conditions, Heart Diseases, Lung Diseases, News, Regulations, Treatment
FDA Approves New Oral Drug for Hereditary Angioedema (HAE)
The U.S. Food and Drug Administration (FDA) has… Oct 8, 2020| Posted in: Congenital And Genetic Conditions, Heart Diseases, Metabolic Disorders, Neurology/Nervous System Diseases
Triheptanoin Approved to Treat Long-chain Fatty Acid Oxidation Disorders
Erik Harris, Executive Vice President and Chief Commercial… Oct 5, 2020| Posted in: Advocacy, Congenital And Genetic Conditions, Heart Diseases, Musculoskeletal Diseases, Neurology/Nervous System Diseases, Ophthalmology/Eye Diseases
Video Game Tournament Provides Support for Duchenne Patients
Tiffany Cook, Family Resource Manager of CureDuchenne, discusses… 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 28, 2020| Posted in: Drug Development, Heart Diseases, Hematologic Disorders, News, Regulations, Treatment
FDA Approves New Treatment for Hypereosinophilic Syndrome
The U.S. Food and Drug Administration (FDA) has… Sep 16, 2020| Posted in: Congenital And Genetic Conditions, Expert Perspectives, Heart Diseases, Lysosomal Storage Disorders, Metabolic Disorders, Musculoskeletal Diseases, Neurology/Nervous System Diseases, Ophthalmology/Eye Diseases