N omenclature of genetic movement disorders: R ecommendations of the international P arkinson and movement disorder society task force

C Marras, A Lang, BP van de Warrenburg… - Movement …, 2016‏ - Wiley Online Library
The system of assigning locus symbols to specify chromosomal regions that are associated
with a familial disorder has a number of problems when used as a reference list of …

Consensus guideline for the diagnosis and treatment of tetrahydrobiopterin (BH4) deficiencies

T Opladen, E López-Laso… - Orphanet journal of rare …, 2020‏ - Springer
Background Tetrahydrobiopterin (BH 4) deficiencies comprise a group of six rare
neurometabolic disorders characterized by insufficient synthesis of the monoamine …

Monoamine neurotransmitter disorders—clinical advances and future perspectives

J Ng, A Papandreou, SJ Heales… - Nature Reviews Neurology, 2015‏ - nature.com
The monoamine neurotransmitter disorders are important genetic syndromes that cause
disturbances in catecholamine (dopamine, noradrenaline and adrenaline) and serotonin …

Dopa-responsive dystonia—clinical and genetic heterogeneity

S Wijemanne, J Jankovic - Nature reviews neurology, 2015‏ - nature.com
Dopa-responsive dystonia (DRD) encompasses a group of clinically and genetically
heterogeneous disorders that typically manifest as limb-onset, diurnally fluctuating dystonia …

Redefining cerebral palsies as a diverse group of neurodevelopmental disorders with genetic aetiology

CL van Eyk, MC Fahey, J Gecz - Nature Reviews Neurology, 2023‏ - nature.com
Cerebral palsy is a clinical descriptor covering a diverse group of permanent, non-
degenerative disorders of motor function. Around one-third of cases have now been shown …

Medical treatment of dystonia

J Jankovic - Movement disorders, 2013‏ - Wiley Online Library
Medications such as anticholinergic drugs, dopamine modulators, baclofen, muscle
relaxants, and other pharmacologic agents have been used for a long time to treat dystonia …

Genetic or other causation should not change the clinical diagnosis of cerebral palsy

AH MacLennan, S Lewis… - Journal of child …, 2019‏ - journals.sagepub.com
High throughput sequencing is discovering many likely causative genetic variants in
individuals with cerebral palsy. Some investigators have suggested that this changes the …

An international survey of patients with tetrahydrobiopterin deficiencies presenting with hyperphenylalaninaemia

T Opladen, GF Hoffmann, N Blau - Journal of inherited metabolic disease, 2012‏ - Springer
Objectives The present study summarizes clinical and biochemical findings, current
treatment strategies and follow-up in patients with tetrahydrobiopterin (BH 4) deficiencies …

A diagnostic approach for cerebral palsy in the genomic era

RW Lee, A Poretti, JS Cohen, E Levey, H Gwynn… - Neuromolecular …, 2014‏ - Springer
An ongoing challenge in children presenting with motor delay/impairment early in life is to
identify neurogenetic disorders with a clinical phenotype, which can be misdiagnosed as …

The genetics of dystonia: new twists in an old tale

G Charlesworth, KP Bhatia, NW Wood - Brain, 2013‏ - academic.oup.com
Dystonia is a common movement disorder seen by neurologists in clinic. Genetic forms of
the disease are important to recognize clinically and also provide valuable information about …