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

New Oral Drug for Patients With Acromegaly

Susan Samson, MD, PhD discusses a new treatment…

CME Program: Gaucher Disease Highlights from WORLDSymposium 2020

Neal Weinreb, MD, FACP highlights the latest information about Gaucher disease from WORLDSymposium and provides expert analysis of its clinical relevance to help health care providers better care for patients with this rare disease.

CME Program: Fabry Disease Highlights from WORLDSymposium 2020

Ozlem Goker-Alpan, MD highlights the latest Fabry disease information from the WORLDSymposium and provides expert analysis of its clinical relevance for members of the care team in order to help them care for patients with this rare disease.

CME Program: Mucopolysaccharidoses (MPSs) Highlights from WORLDSymposium 2020

Barbara K. Burton, MD highlights the latest information about MPSs from WORLDSymposium and provides expert analysis of its clinical relevance for members of the care team in order to help them care for patients with these rare diseases.

Neurology/Nervous System Diseases

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