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Glycogen storage disease type III (GSD III) is a rare genetic disorder resulting from mutations in the AGL gene, which encodes the glycogen debranching enzyme (GDE).
Glycogen storage disease type III (GSD III) is an autosomal recessive disease caused by the deficiency of glycogen-debranching enzyme (AGL). The overall incidence of the disease is about 1:100,000 ...
The paper identifies a mutation in the gene encoding the glycogen debranching enzyme (AGL), which had previously been undetected in a decade of investigation by the same authors.
Notably, GSDIII is caused by a glycogen-debranching enzyme deficiency resulting in glycogen accumulation in the liver and muscle. The company plans to begin enrollment in a phase I/II study ...
Glycogen Storage Disease Type III, or GSDIII, a disease caused by a glycogen debranching enzyme (AGL) deficiency that results in glycogen accumulation in the liver and muscle. GSDIII can cause ...
The paper identifies a mutation in the gene encoding the glycogen debranching enzyme (AGL), which had previously been undetected in a decade of investigation by the same authors.
Phase 1/2 study is expected to initiate enrollment in the second half of 2021NOVATO, Calif., July 27, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical ...
GLYCOGEN STORAGE DISEASE TYPE III (GSD III) was diagnosed in 4 Inuit children (3 confirmed, 1 suspected case) at our institution over the last decade. This rare autosomal recessive disease, which ...
--Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced ...
Ultragenyx Pharmaceutical Inc. RARE announced that the FDA has cleared the investigational new drug or IND application, for its lead mRNA therapy, UX053, which is being developed for the treatment ...
The paper identifies a mutation in the gene encoding the glycogen debranching enzyme (AGL), which had previously been undetected in a decade of investigation by the same authors.