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Pendred syndrome

ORPHA:705Malformation syndromeAutosomal recessiveInfancy, Neonatal

Ассоциированные гены (3)

SLC26A4
solute carrier family 26 member 4
Disease-causing germline mutation(s) in
OMIM: 605646
KCNJ10
potassium inwardly rectifying channel subfamily J member 10
Disease-causing germline mutation(s) in
OMIM: 602208
FOXI1
forkhead box I1
Disease-causing germline mutation(s) in
OMIM: 601093

Фенотипы (15)

Очень частый (80–99%)4
HP:0000359Abnormality of the inner ear
HP:0000407Sensorineural hearing impairment
HP:0008586Hypoplasia of the cochlea
HP:0011387Enlarged vestibular aqueduct
Частый (30–79%)2
HP:0000821Hypothyroidism
HP:0000853Goiter
Периодический (5–29%)9
HP:0000112Nephropathy
HP:0000843Hyperparathyroidism
HP:0001249Intellectual disability
HP:0001251Ataxia
HP:0002093Respiratory insufficiency
HP:0002167Abnormality of speech or vocalization
HP:0002321Vertigo
HP:0002777Tracheal stenosis
HP:0002890Thyroid carcinoma

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