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Athyreosis

ORPHA:95713Morphological anomalyAutosomal dominantInfancy, Neonatal

Ассоциированные гены (6)

FOXE1
forkhead box E1
Candidate gene tested in
OMIM: 602617
NKX2-1
NK2 homeobox 1
Candidate gene tested in
OMIM: 600635
SLC26A4
solute carrier family 26 member 4
Disease-causing germline mutation(s) (loss of function) in
OMIM: 605646
TSHR
thyroid stimulating hormone receptor
Disease-causing germline mutation(s) (loss of function) in
OMIM: 603372
NKX2-5
NK2 homeobox 5
Disease-causing germline mutation(s) in
OMIM: 600584
PAX8
paired box 8
Disease-causing germline mutation(s) (loss of function) in
OMIM: 167415

Фенотипы (25)

Очень частый (80–99%)14
HP:0000158Macroglossia
HP:0000239Large fontanelles
HP:0000271Abnormality of the face
HP:0000280Coarse facial features
HP:0000821Hypothyroidism
HP:0001252Hypotonia
HP:0001324Muscle weakness
HP:0002019Constipation
HP:0003270Abdominal distention
HP:0008191Thyroid agenesis
HP:0011968Feeding difficulties
HP:0012378Fatigue
HP:0025483Abnormal circulating thyroglobulin concentration
HP:0100786Hypersomnia
Частый (30–79%)11
HP:0000282Facial edema
HP:0001254Lethargy
HP:0001263Global developmental delay
HP:0001510Growth delay
HP:0001537Umbilical hernia
HP:0001615Hoarse cry
HP:0004322Short stature
HP:0006579Prolonged neonatal jaundice
HP:0008282Unconjugated hyperbilirubinemia
HP:0010864Intellectual disability, severe
HP:0033850Coldness

Эпидемиология (1)

Point prevalence
1-9 / 100 000
Europe

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы