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Rare Disease Institute in Children’s National Hospital now is seeing patients at their their new location on the Research & Innovation Campus, 7125 13th Pl NW Washington, DC 20012.

April 20, 2021 December 8, 2021 By childrenraredisease
CDC

Rare Disease Institute - Genetics and Metabolism | Children's National Hospital

FDA Approves First Treatment for Rare Genetic Metabolic Pediatric Disorder (ajmc.com)

April 21, 2020 December 8, 2021 By childrenraredisease
CDC

FDA Approves First Treatment for Rare Genetic Metabolic Pediatric Disorder (ajmc.com) The disorder known as molybdenum cofactor deficiency (MoCD) Type A presents shortly after birth, often with severe encephalopathy and intractable seizures. The FDA approved the first therapy for an ultra-rare genetic metabolic disorder that affects infants, who don’t normally live past the age of 4. The disorder is...

branding

Rare Disease Institute in Children’s National Hospital now is seeing patients at their their new location on the Research & Innovation Campus, 7125 13th Pl NW Washington, DC 20012.

FDA Approves First Treatment for Rare Genetic Metabolic Pediatric Disorder (ajmc.com)

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