Regulations
Jun 22, 2022| Posted in: Advocacy, Autoimmune / Autoinflammatory Disorders, Cancers, Congenital And Genetic Conditions, Diagnosis, 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, Ophthalmology/Eye Diseases, Regulations, Skin Conditions, Treatment
New Report Estimates the Number of Rare Diseases Is More Than 10,000
Kirk Lamoreaux, discusses ‘The Power of Being Counted,’… Jun 17, 2022| Posted in: Congenital And Genetic Conditions, Drug Development, Endocrine Disorders, Neurology/Nervous System Diseases, Regulations, Treatment
FDA Approves First-Ever Treatment for Patients with Bardet-Biedl Syndrome
Bardet-Biedl Syndrome is a rare genetic disease that… Jun 16, 2022| Posted in: Advocacy, Autoimmune / Autoinflammatory Disorders, Cancers, Congenital And Genetic Conditions, Diagnosis, Drug Development, Endocrine Disorders, Expert Perspectives, Gastrointestinal Diseases, Heart Diseases, Hematologic Disorders, Kidney And Urinary Diseases, Lung Diseases, Lysosomal Storage Disorders, Metabolic Disorders, Musculoskeletal Diseases, Neurology/Nervous System Diseases, Ophthalmology/Eye Diseases, Regulations, Skin Conditions, Treatment
NORD’s Rare Disease Centers of Excellence Benefits Individuals and Communities
Dr. Priya Kishnani at Duke University School of… Jun 14, 2022| Posted in: Congenital And Genetic Conditions, Drug Development, Neurology/Nervous System Diseases, Regulations, Treatment
FDA Approves Vutrisiran to Treat Neuropathy in Hereditary ATTR Amyloidosis
FDA approves Alnylam’s RNAi drug for polyneuropathy in… May 31, 2022| Posted in: Congenital And Genetic Conditions, Diagnosis, Gastrointestinal Diseases, Metabolic Disorders, Regulations, Treatment
NICE Recommends Odevixibat for Progressive Familial Intrahepatic Cholestasis (PFIC)
Dr. Diedre Kelly summarizes the data presented to… May 25, 2022| Posted in: Advocacy, Autoimmune / Autoinflammatory Disorders, Cancers, Congenital And Genetic Conditions, Diagnosis, 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, Ophthalmology/Eye Diseases, Regulations, Skin Conditions, Treatment
11 Rare Disease Research Working Groups In Development
Dr Edward Neilan of NORD discusses how the… May 23, 2022| Posted in: Advocacy, Autoimmune / Autoinflammatory Disorders, Cancers, Congenital And Genetic Conditions, Diagnosis, Drug Development, Endocrine Disorders, Expert Perspectives, Gastrointestinal Diseases, Heart Diseases, Hematologic Disorders, Kidney And Urinary Diseases, Lung Diseases, Lysosomal Storage Disorders, Metabolic Disorders, Musculoskeletal Diseases, Neurology/Nervous System Diseases, Ophthalmology/Eye Diseases, Regulations, Skin Conditions, Treatment
NORD’s Centers of Excellence Addresses Unmet Medical Needs
As Dr. Bodamer explains, one of the goals… May 21, 2022| Posted in: Autoimmune / Autoinflammatory Disorders, Drug Development, Gastrointestinal Diseases, Regulations, Treatment
FDA Approves First-Ever Treatment for Eosinophilic Esophagitis (EoE)
EoE is a chronic, allergic inflammatory disease due… May 14, 2022| Posted in: Drug Development, Neurology/Nervous System Diseases, Regulations, Treatment
FDA Approves Oral Formulation of Edaravone to Treat ALS
The oral formulation can be administered orally or… May 10, 2022| Posted in: Advocacy, Autoimmune / Autoinflammatory Disorders, Cancers, Congenital And Genetic Conditions, Diagnosis, 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, Ophthalmology/Eye Diseases, Regulations, Skin Conditions, Treatment