Drug Development
Jun 29, 2022| Posted in: Drug Development, Endocrine Disorders
AZP-3601 in Development to Treat Hypoparathyroidism
Dr. Thierry Abribat, CEO of Amolyt Pharmaceuticals, explains… Jun 24, 2022| Posted in: Congenital And Genetic Conditions, Drug Development
NCATS’ “Many-Diseases-At-A-Time” Approach to Gene Therapy
Dr. P.J. Brooks, Acting Director of the Office… 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 21, 2022| Posted in: Congenital And Genetic Conditions, Drug Development, Neurology/Nervous System Diseases
RNA-Based Therapy Shows Promise For Dravet Syndrome in Preclinical Studies
Dr. Ann Barbier of CAMP4 Therapeutics, discusses positive… Jun 21, 2022| Posted in: Congenital And Genetic Conditions, Diagnosis, Drug Development, Lysosomal Storage Disorders, Neurology/Nervous System Diseases
Positive Interim Data in Gene Therapy Trial for GM1 Gangliosidosis
Dr. Mark Forman, Chief Medical Officer at Passage… 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… Jun 13, 2022| Posted in: Cancers, Diagnosis, Drug Development, Hematologic Disorders, Treatment
Positive Updated Results from MajesTEC-1 Study in Relapsed/Refractory Multiple Myeloma
Dr. Ajay Nooka, of the Emory School of… Jun 10, 2022| Posted in: Congenital And Genetic Conditions, Drug Development, Neurology/Nervous System Diseases, Treatment