autosomal recessive dopa-responsive dystonia

ORPHA: 101150

Overview

Autosomal recessive dopa-responsive dystonia (DYT5b) is a very rare neurometabolic disorder characterized by a spectrum of symptoms ranging from those seen in dopa-responsive dystonia (DRD; see this term) to progressive infantile encephalopathy

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 autosomal recessive dopa-responsive dystonia, sourced from HPO and Orphanet clinical annotations.

PtosisIrritabilityDelayed speech and language developmentAtaxiaHypotoniaLethargyMotor delayParkinsonismMyoclonusBrisk reflexesPes cavusTalipes equinovarusConstipationRigidityGait ataxiaBradykinesiaAbnormality of extrapyramidal motor functionPostural tremorHypokinesiaLower limb hyperreflexiaLimb dystoniaBabinski signExcessive salivationDecreased CSF homovanillic acid concentrationFocal dystoniaOculogyric crisisFeeding difficultiesNight sweatsMild intellectual disabilityFeverGeneralized dystoniaGeneralized hypotoniaProgressive encephalopathy

Classification & Codes

Orphanet Code

ORPHA:101150
autosomal recessive dopa-responsive dystonia
OrphanetORPHA:101150
Treatments0 drug(s)
Symptoms on record33 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO
autosomal recessive dopa-responsive dystonia | OrphanDrug