Neurology/Nervous System Diseases
Jan 18, 2021| Posted in: Congenital And Genetic Conditions, Diagnosis, Lysosomal Storage Disorders, Metabolic Disorders, Neurology/Nervous System Diseases
Diagnosing Niemann-Pick Type C
Elizabeth Berry-Kravis, MD, PhD, co-director of the Molecular… Jan 15, 2021| Posted in: Congenital And Genetic Conditions, Diagnosis, Lysosomal Storage Disorders, Metabolic Disorders, Neurology/Nervous System Diseases
Overview of Niemann-Pick Type C
Elizabeth Berry-Kravis, MD, PhD, co-director of the Molecular… Jan 13, 2021| Posted in: Congenital And Genetic Conditions, Neurology/Nervous System Diseases
Huntington Disease – The Worst Disease Known to Humans
Michael Hayden, PhD, Chief Executive Officer of Prilenia Therapeutics,… Jan 12, 2021| Posted in: Congenital And Genetic Conditions, Heart Diseases, Hematologic Disorders, Lysosomal Storage Disorders, Metabolic Disorders, Neurology/Nervous System Diseases, Ophthalmology/Eye Diseases, Skin Conditions, Treatment
Determining the Best Treatment Option for Fabry Disease
Robert Hopkin, MD, Associate Professor of Clinical Pediatrics… Jan 8, 2021| Posted in: Congenital And Genetic Conditions, Heart Diseases, Hematologic Disorders, Kidney And Urinary Diseases, Lysosomal Storage Disorders, Metabolic Disorders, Neurology/Nervous System Diseases, Ophthalmology/Eye Diseases, Skin Conditions
Overview of Fabry Disease
Robert Hopkin, MD, Associate Professor of Clinical Pediatrics… Jan 7, 2021| Posted in: Congenital And Genetic Conditions, Drug Development, Neurology/Nervous System Diseases
PROOF-HD: Pivotal Huntington Disease Trial Underway
Michael Hayden, PhD, Chief Executive Officer of Prilenia Therapeutics,… Jan 5, 2021| Posted in: Advocacy, Congenital And Genetic Conditions, Diagnosis, Metabolic Disorders, Neurology/Nervous System Diseases
Adult-Onset Niemann-Pick Disease: A Diagnostic Journey
Cara Gilmore, a patient with adult onset Niemann-Pick… Jan 4, 2021| Posted in: Autoimmune / Autoinflammatory Disorders, Cancers, Congenital And Genetic Conditions, 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, News, Ophthalmology/Eye Diseases, Skin Conditions, Treatment
Orphan Drugs Currently Under Review by the FDA
Every potential Food and Drug Administration (FDA) approval… Dec 26, 2020| Posted in: Cancers, Congenital And Genetic Conditions, Endocrine Disorders, Musculoskeletal Diseases, Neurology/Nervous System Diseases, Treatment
Acromegaly Guidelines and Treatment Options
Roberto Salvatori, MD, Medical Director, Johns Hopkins Pituitary… Dec 16, 2020| Posted in: Cancers, Congenital And Genetic Conditions, Endocrine Disorders, Metabolic Disorders, Musculoskeletal Diseases, Neurology/Nervous System Diseases, News, Treatment