Metabolic Disorders
Sep 22, 2021| 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, News, Ophthalmology/Eye Diseases, Regulations, Skin Conditions, Treatment
Mary Dunkle of NORD discusses the upcoming Rare…
What the NORD Breakthrough Summit Will Look Like in 2021
Sep 21, 2021| 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, News, Ophthalmology/Eye Diseases, Regulations, Skin Conditions, Treatment
Great conversation with Drs Paul Howard, Eric March,…
CheckRare Panel Discussion: Data Silos Are Hindering Rare Disease Research
Sep 8, 2021| Posted in: Congenital And Genetic Conditions, Lysosomal Storage Disorders, Metabolic Disorders, Neurology/Nervous System Diseases, Ophthalmology/Eye Diseases
Joslyn Crowe, executive director of the National Niemann-Pick…
Niemann-Pick Community Devastated by FDA’s Denial of Approval of Arimoclomol
Sep 3, 2021| Posted in: Autoimmune / Autoinflammatory Disorders, Cancers, Congenital And Genetic Conditions, Drug Development, Endocrine Disorders, Gastrointestinal 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
Rare diseases and orphan drugs are at the…
2021 Orphan Drugs: PDUFA Dates and FDA Approvals
Aug 26, 2021| Posted in: Cancers, Drug Development, Metabolic Disorders, News, Treatment
The Food and Drug Administration (FDA) announced the…
FDA Approves Ivosidenib for the Treatment of Advanced or Metastatic Cholangiocarcinoma
Jul 29, 2021| Posted in: Congenital And Genetic Conditions, Lysosomal Storage Disorders, Metabolic Disorders, Musculoskeletal Diseases, Neurology/Nervous System Diseases, Ophthalmology/Eye Diseases
Sharon Hrynkow, MD, Chief Scientific Officer and Senior…
Mechanism of Action of Hydroxypropyl Betacyclodextrin to Treat Niemann-Pick Disease
Jul 23, 2021| Posted in: Congenital And Genetic Conditions, Drug Development, Lysosomal Storage Disorders, Metabolic Disorders, Musculoskeletal Diseases, Neurology/Nervous System Diseases, Ophthalmology/Eye Diseases
Sharon Hrynkow, MD, Chief Scientific Officer and Senior…
Niemann-Pick Disease Clinical Trial (Phase 3) Enrolling Patients
Jul 20, 2021| Posted in: Congenital And Genetic Conditions, Metabolic Disorders
The Food and Drug Administration (FDA) has approved…
FDA Approves Odevixibat, First Drug Therapy for PFIC
Jun 24, 2021| Posted in: Congenital And Genetic Conditions, Drug Development, Endocrine Disorders, Metabolic Disorders, Musculoskeletal Diseases, Neurology/Nervous System Diseases, Ophthalmology/Eye Diseases
Richard Colvin, MD, PhD, Vice President of Clinical…
CHMP Recommends Marketing Authorization of Gene Therapy for Early CALD
Jun 18, 2021| Posted in: Congenital And Genetic Conditions, Lysosomal Storage Disorders, Metabolic Disorders, News
The U.S. Food and Drug Administration (FDA) has…
FDA Denies Approval of Arimoclomol for the Treatment of Niemann-Pick Type C
