Neurology/Nervous System Diseases

Jul 28, 2020| Posted in: Congenital And Genetic Conditions, Diagnosis, Gastrointestinal Diseases, Heart Diseases, Hematologic Disorders, Kidney And Urinary Diseases, Lysosomal Storage Disorders, Metabolic Disorders, Neurology/Nervous System Diseases, Ophthalmology/Eye Diseases, Skin Conditions

Fabry Disease and the Heart

Ozlem Goker-Alpan, MD, provides a summary of research…

Jul 27, 2020| Posted in: Congenital And Genetic Conditions, Drug Development, Musculoskeletal Diseases, Neurology/Nervous System Diseases, News

Update on the Phase 2 Facioscapulohumeral Muscular Dystrophy Clinical Trial

Diego Cadavid, MD of Fulcrum Therapeutics provides an…

Jul 23, 2020| Posted in: Congenital And Genetic Conditions, Neurology/Nervous System Diseases, News, Treatment

FDA Approval of Fintepia (fenfluramine) to Treat Dravet Syndrome

Ashish Sagrolikar, Chief Commercial Officer of Zogenix, discusses…

Jul 21, 2020| Posted in: Congenital And Genetic Conditions, Drug Development, Musculoskeletal Diseases, Neurology/Nervous System Diseases, News

Research Presented at AAN 2020 Show Promise for FSHD Treatment

Diego Cadavid, MD, of Fulcrum Therapeutics summarizes the…

Jul 15, 2020| Posted in: Congenital And Genetic Conditions, Diagnosis, Drug Development, Neurology/Nervous System Diseases

Dravet Syndrome Explained: “A Constant State of High Alert”

Ashish Sagrolikar, chief commercial officer of Zogenix, discusses…

Jul 5, 2020| Posted in: Diagnosis, Gastrointestinal Diseases, Musculoskeletal Diseases, Neurology/Nervous System Diseases, Ophthalmology/Eye Diseases

Tips for Diagnosing Neuromyelitis Optica Spectrum Disorder (NMOSD)

Bruce Cree, MD of the University of California…

Jun 30, 2020| Posted in: Neurology/Nervous System Diseases, Ophthalmology/Eye Diseases, Treatment

Approved Treatments for Neuromyelitis Optica Spectrum Disorder

Bruce Cree, MD of the University of California…

Jun 26, 2020| Posted in: Autoimmune / Autoinflammatory Disorders, Drug Development, Musculoskeletal Diseases, Neurology/Nervous System Diseases, News, Regulations, Treatment

FDA Approves Fenfluramine to Reduce Seizures in Children with Dravet Syndrome

The U.S. Food and Drug Administration has approved…

Jun 23, 2020| Posted in: Advocacy, Congenital And Genetic Conditions, Diagnosis, Metabolic Disorders, Neurology/Nervous System Diseases, Ophthalmology/Eye Diseases

Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency

Aromatic L-amino acid decarboxylase (AADC) deficiency is a…

Jun 23, 2020| Posted in: Autoimmune / Autoinflammatory Disorders, Musculoskeletal Diseases, Neurology/Nervous System Diseases, Ophthalmology/Eye Diseases

New Studies Show Continued Benefit of Satralizumab to Treat NMOSD

Kathleen Hawker, MD at Genentech describes two abstracts…

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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