First Patient Receives Gene Therapy For Rare Liver Disorder
Worlds First Gene Therapy Clinical Trial For Glycogen Storage Disease Approved By FDA
Rare disease group urges Ontario to include devastating neuromuscular condition in newborn screening program (Newswire.ca)
Dimension Therapeutics to Develop Gene Therapy Treatments for Patients with Ornithine Transcarbamylase (OTC) Deficiency and Glycogen Storage Disease Type Ia (BusinessWire.com)
Glycogen storage disease could affect 1 in 500 Nunavik Inuit (CBC.ca)
Boy's 'Chocolate Bar' Book Nets $1M for Rare Liver Disorder Research (ABCnews.go.com)
Minnesota mom: A rare glycogen storage disease (StarTribune.com)
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