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Entries by childrenraredisease
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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.
Rare Disease Institute - Genetics and Metabolism | Children's National Hospital
National Organization for Rare Disorders (NORD) will host Rare Disease Breakthrough Summit on Oct 18-19 this year.
Please check Summit Event Details - NORD (National Organization for Rare Disorders) (rarediseases.org) for details.
Rare Diseases and Orphan Products Breakthrough Summit October 18-19, 2021
2020 Summit Pricing
*2021 Pricing and dates subject to change
Early Bird (ends 8/27/2021)
Standard
NORD Patient Organization Members
$185
$345
Registered 501c3 Non-Profits/Patients
$213
$370
Academic/Physician
$213
$370
NORD Corporate Council Members
$900
$1,112
Industry (Pharma, Service Providers, Co-Pay Foundations)
$1,165
$1,325
Government
$249
$249
Pricing includes all online content on Monday,...
You can search any pediatric rare disease designation at FDA database
Search Orphan Drug Designations and Approvals (fda.gov)
FDA Approves First Treatment for Rare Genetic Metabolic Pediatric Disorder (ajmc.com)
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...