← Назад

Deafness-enamel hypoplasia-nail defects syndrome

ORPHA:3220Malformation syndromeAutosomal recessiveChildhood

Ассоциированные гены (2)

PEX1
peroxisomal biogenesis factor 1
Disease-causing germline mutation(s) (loss of function) in
OMIM: 602136
PEX6
peroxisomal biogenesis factor 6
Disease-causing germline mutation(s) (loss of function) in
OMIM: 601498

Фенотипы (33)

Очень частый (80–99%)18
HP:0001231Abnormal fingernail morphology
HP:0001249Intellectual disability
HP:0003241External genital hypoplasia
HP:0003777Pili torti
HP:0008388Abnormal toenail morphology
HP:0011362Abnormal hair quantity
HP:0045074Thin eyebrow
HP:0100643Abnormality of nail color
HP:0000135Hypogonadism
HP:0000164Abnormality of the dentition
HP:0000365Hearing impairment
HP:0000407Sensorineural hearing impairment
HP:0000492Abnormal eyelid morphology
HP:0000534Abnormal eyebrow morphology
HP:0000614Abnormal nasolacrimal system morphology
HP:0000679Taurodontia
HP:0000682Abnormality of dental enamel
HP:0000819Diabetes mellitus
Частый (30–79%)5
HP:0000311Round face
HP:0000786Primary amenorrhea
HP:0001176Large hands
HP:0004322Short stature
HP:0011675Arrhythmia
Периодический (5–29%)10
HP:0000763Sensory neuropathy
HP:0000956Acanthosis nigricans
HP:0001265Hyporeflexia
HP:0002514Cerebral calcification
HP:0002750Delayed skeletal maturation
HP:0008064Ichthyosis
HP:0009830Peripheral neuropathy
HP:0009890High anterior hairline
HP:0010547Muscle flaccidity
HP:0100490Camptodactyly of finger

Эпидемиология (2)

Cases/families
Worldwide
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