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Amelocerebrohypohidrotic syndrome

ORPHA:1946Malformation syndromeAutosomal recessiveChildhood, Infancy, Neonatal

Ассоциированные гены (2)

SLC13A5
solute carrier family 13 member 5
Disease-causing germline mutation(s) in
OMIM: 608305
ROGDI
rogdi atypical leucine zipper
Disease-causing germline mutation(s) (loss of function) in
OMIM: 614574

Фенотипы (14)

Очень частый (80–99%)11
HP:0001250Seizure
HP:0001257Spasticity
HP:0001268Mental deterioration
HP:0002353EEG abnormality
HP:0002376Developmental regression
HP:0006286Yellow-brown discoloration of the teeth
HP:0011073Abnormality of dental color
HP:0000682Abnormality of dental enamel
HP:0000705Amelogenesis imperfecta
HP:0000726Dementia
HP:0010864Intellectual disability, severe
Частый (30–79%)1
HP:0000966Hypohidrosis
Периодический (5–29%)2
HP:0000238Hydrocephalus
HP:0004322Short stature

Эпидемиология (1)

Point prevalence
<1 / 1 000 000
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы