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Phosphoglycerate Kinase Deficiency

Systemic/MultisystemOMIM 311800
W
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Overview

From WUSTL Neuromuscular

● Genetics Mutation types: Missense, Deletion & Splice site Mutation locations: All along gene except myopathic form > 20 mutations described Protein Ubiquitous expression except in sperm Glycolytic pathway Generates ATP through conversion of 1,3-DPG to 3-PGA Clinical features: Only rarely (6%) all in same patient Hemolytic anemia (HNSHA) (34%): Non-spherocytic CNS dysfunction Mental retardation Seizures Behavioral abnormalities Strokes Usually associated with anemia Muscle Myopathy (Slowly progressive) Cramps Myalgia: Exercise induced ± Myoglobinuria Laboratory Serum CK: High or Normal Ischem

OMIM Entries

OMIM #311800

Related Conditions

Shared genes
Granulomatous & Inflammatory Myopathies with Anti-mitochondrial antibodies
M2E2CLINICAL
IMPP (Myofasciitis; EVIM) + Deafness: Enterovirus & Hypogammaglobulinemia-Related
D68D71CLINICAL
Vici syndrome
IICLINICAL

External Resources

WUSTL Neuromuscular
Washington University Disease Center
OMIM
Online Mendelian Inheritance in Man
PubMed
Biomedical Literature
GeneReviews
NCBI Expert-Authored Reviews
Orphanet
Portal for Rare Diseases
NORD
National Organization for Rare Disorders

Data sourced from the Washington University Neuromuscular Disease Center. For clinical use, always refer to primary sources.