Neurology/Nervous System Diseases
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 10, 2022| Posted in: Congenital And Genetic Conditions, Drug Development, Neurology/Nervous System Diseases, Treatment
RNA-Based Therapy For Dravet Syndrome
As Dr. Barbier of CAMP4 Therapeutics explains, CMP-SCN… Jun 1, 2022| Posted in: Congenital And Genetic Conditions, Diagnosis, Lysosomal Storage Disorders, Musculoskeletal Diseases, Neurology/Nervous System Diseases
What Is Alpha Mannosidosis?
Dr. Barbara Burton of the Lurie Children’s Hospital… May 30, 2022| Posted in: Congenital And Genetic Conditions, Diagnosis, Drug Development, Lysosomal Storage Disorders, Musculoskeletal Diseases, Neurology/Nervous System Diseases
An Interview With Dr. Raymond Wang About Mucopolysaccharidosis Type I (MPS I)
Dr. Raymond Wang talks to CheckRare about mucopolysaccharidosis… May 27, 2022| Posted in: Advocacy, Congenital And Genetic Conditions, Diagnosis, Neurology/Nervous System Diseases