Helpful resources for Rare Disorders

Boston Children’s Hospital

European Medicines Agency (EMA)

US Food and Drug Administration (FDA)

Genomics Research and Innovation Network (GRIN)

National Center for Biotechnology Information (NCBI)

National Organization for Rare Disorders (NORD)



Disease-specific Organizations and Foundations

All Things Kabuki (ATK)

National MPS Society

National Niemann-Pick Disease Foundation Inc. (NNPDF)

Organic Acidemia Association (OAA)

National Tay-Sachs & Allied Diseases (NTSAD)