Endocrine Disorders
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 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: Advocacy, Congenital And Genetic Conditions, Endocrine Disorders, Musculoskeletal Diseases
Newly-Diagnosed ENPP1 Deficiency: Advice for Parents
Axel Bolte, CEO of Inozyme Pharmaceuticals, gives advice… 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… 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 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
Rare Diseases Cost 10 Times More Than Common Diseases
The overall cost estimate for all rare disease… May 6, 2022| Posted in: Congenital And Genetic Conditions, Drug Development, Endocrine Disorders, Musculoskeletal Diseases
CEO of Inozyme Shares Preliminary Data From ENPP1 Deficiency Clinical Trial
Axel Bolte, CEO of Inozyme Pharmaceuticals, shares early… Apr 21, 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, Skin Conditions, Treatment