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Developmental delay-language impairment-dopa responsive dystonia-parkinsonism syndrome due to 2q24 microdeletion

ORPHA:1617Etiological subtypeNot applicableAntenatal, Neonatal

Ассоциированные гены (2)

NR4A2
nuclear receptor subfamily 4 group A member 2
Role in the phenotype of
OMIM: 601828
TBR1
T-box brain transcription factor 1
Candidate gene tested in
OMIM: 604616

Фенотипы (28)

Очень частый (80–99%)19
HP:0000175Cleft palate
HP:0000470Short neck
HP:0000494Downslanted palpebral fissures
HP:0000525Abnormality iris morphology
HP:0000708Atypical behavior
HP:0001188Hand clenching
HP:0001249Intellectual disability
HP:0001250Seizure
HP:0001263Global developmental delay
HP:0001319Neonatal hypotonia
HP:0001508Failure to thrive
HP:0001510Growth delay
HP:0001518Small for gestational age
HP:0001770Toe syndactyly
HP:0010078Bullet-shaped distal phalanx of the hallux
HP:0011344Severe global developmental delay
HP:0100490Camptodactyly of finger
HP:0100807Long fingers
HP:0000358Posteriorly rotated ears
Частый (30–79%)8
HP:0000190Abnormal oral frenulum morphology
HP:0000274Small face
HP:0000316Hypertelorism
HP:0000322Short philtrum
HP:0000518Cataract
HP:0000568Microphthalmia
HP:0000589Coloboma
HP:0000729Autistic behavior
Периодический (5–29%)1
HP:0002871Central apnea

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