Long-term course of L-dopa-responsive dystonia caused by tyrosine hydroxylase deficiency.

Schiller, A; Wevers, R A; Steenbergen, G C H; Blau, N; Jung, H H · Neurology · 2004

case_series · Level IV

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Abstract

The authors report the long-term course of two siblings with L-dopa responsive dystonia (DRD) associated with a compound heterozygous mutation in the tyrosine hydroxylase (TH) gene. Both siblings manifested with lower-limb onset generalized DRD and had a sustained response to low-dose L-dopa therapy for over 35 years. Although the l-dopa therapy was delayed up to 20 years after disease onset, there were no cognitive or neurologic sequelae of the long-term catecholamine deficit.

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