Pitt-Hopkins syndrome

MeSH: C537403ORPHA: 2896

Overview

rare genetic disorder characterized by developmental delay, epilepsy, distinctive facial features, and possible intermittent hyperventilation followed by apnea

Available Treatments (0)

No treatments linked yet

Clinical trials or compassionate use may be available — consult a specialist.

Clinical Presentation

Signs and symptoms associated with Pitt-Hopkins syndrome, sourced from HPO and Orphanet clinical annotations.

Wide mouthAbnormal palate morphologyMicrocephalyCoarse facial featuresFull cheeksShort philtrumNarrow foreheadThickened helicesProminent nasal bridgeAnteverted naresShort neckAstigmatismDeeply set eyeMyopiaUpslanted palpebral fissureTooth malpositionSingle transverse palmar creaseTapered fingerIntellectual disabilityAtaxiaHypotoniaGlobal developmental delaySpecific learning disabilityFailure to thriveGrowth delayPes planusConstipationGastroesophageal refluxHiatus herniaMutismModerate intellectual disabilitySleep disturbanceAphasiaFailure of eruption of permanent teethPes valgusEcholaliaShort metatarsalAbnormal helix morphologyBroad fingertipOverhanging nasal tipFeeding difficultiesThick vermilion borderFinger clinodactylyEsophagitisSmall handTriangular nasal tipStrabismusAcrocyanosisSeizureAbsent speechNarrow footGait ataxiaSmall cerebral cortexAbnormal pattern of respirationHyperventilationAplasia/Hypoplasia of the corpus callosumSleep apneaCryptorchidismMicropenisAggressive behaviorAutistic behaviorHypopigmented skin patchesSupernumerary nippleScoliosisPostnatal growth retardationHappy demeanorSelf-injurious behaviorAganglionic megacolonHodgkin lymphoma

Classification & Codes

MeSH Code

C537403

Orphanet Code

ORPHA:2896
Pitt-Hopkins syndrome
MeSHC537403
OrphanetORPHA:2896
Treatments0 drug(s)
Symptoms on record69 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO