Diseases

Spotlight on rare and genetic diseases and cancers

Accelerating the Road to Treatment: The Global Commission to End the Diagnostic Odyssey for Children
Mucopolysaccharidosis (MPS) Resource Center
Rare Disease Day 2019
Acid Sphingomyelinase Deficiency (ASMD)
Acute Myeloid Leukemia
Bile Acid Synthesis
Cerebrotendinous Xanthomatosis
Charcot-Marie-Tooth Disease – Type 1A
Congenital Myasthenic Syndromes
Cystinuria
Cushing Syndrome
Duchenne Muscular Dystrophy
Eosinophilic Granulomatosis With Polyangiitis (EPGA)
Fabry Disease
Focal Segmental Glomerulosclerosis
Hereditary ATTR Amyloidosis
Gaucher Disease
Lambert-Eaton Myasthenic Syndrome
Lysosomal Storage Disorders
Pantothenate Kinase-Associated Neurodegeneration (PKAN)
Peroxisome Biogenesis Disorder (Zellweger Spectrum Disorders)
Pompe Disease (also called Glycogen storage disease type 2)
Pulmonary Arterial Hypertension
Spinal Muscular Atrophy
Tenosynovial Giant Cell Tumor (TGCT)
Zellweger Syndrome

NECESSARY CONVERSATIONS: Cardiac Amyloidosis and Other Rare Diseases Impacting the Health of Our Communities - Yahoo Finance February 28, 2020
Rare friendship: Shared diagnosis bonds young Wooster ’besties’ - Wooster Daily Record February 28, 2020
Following sister’s death, Lauren Surrey loses battle with same rare disease - SILive.com February 28, 2020
Hyderabad observes World Rare Disease Day - Big News Network February 28, 2020
Families come together to mark Rare Disease Day in Hartford - WTNH.com February 28, 2020

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