Kees Okkersen
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View article: Clinical improvement of DM1 patients reflected by reversal of disease-induced gene expression in blood
Clinical improvement of DM1 patients reflected by reversal of disease-induced gene expression in blood Open
View article: Clinical Outcome Evaluations and CBT Response Prediction in Myotonic Dystrophy
Clinical Outcome Evaluations and CBT Response Prediction in Myotonic Dystrophy Open
Background: The European OPTIMISTIC clinical trial has demonstrated a significant, yet heterogenous effect of Cognitive Behavioural Therapy (CBT) for Myotonic Dystrophy type 1 (DM1) patients. One of its remaining aims was the assessment of…
View article: Associations Between Variant Repeat Interruptions and Clinical Outcomes in Myotonic Dystrophy Type 1
Associations Between Variant Repeat Interruptions and Clinical Outcomes in Myotonic Dystrophy Type 1 Open
ClinicalTrials.gov NCT02118779.
View article: Clinical outcome evaluations and CBT response prediction in Myotonic Dystrophy
Clinical outcome evaluations and CBT response prediction in Myotonic Dystrophy Open
Background The European OPTIMISTIC clinical trial has demonstrated a significant, yet heterogenous effect of Cognitive Behavioural Therapy (CBT) for Myotonic Dystrophy type 1 (DM1) patients. One of its remaining aims was the assessment of …
View article: Human brain pathology in myotonic dystrophy type 1: A systematic review
Human brain pathology in myotonic dystrophy type 1: A systematic review Open
Brain involvement in myotonic dystrophy type 1 (DM1) is characterized by heterogeneous cognitive, behavioral, and affective symptoms and imaging alterations indicative of widespread grey and white matter involvement. The aim of the present…
View article: MYOTONIC DYSTROPHY TYPE 1
MYOTONIC DYSTROPHY TYPE 1 Open
Myotonic dystrophy type 1 is an autosomal dominant multisystem disease caused by a trinucleotide repeat expansion in the DMPK gene on chromosome 19. Named after its neuromuscular involvement, muscle weakness and myotonia are often key char…
View article: Characterization of EEG-based functional brain networks in myotonic dystrophy type 1
Characterization of EEG-based functional brain networks in myotonic dystrophy type 1 Open
This work was supported by the European Community's Seventh Framework Programme (FP7/2007-2013) under grant agreement n° 305697 (OPTIMISTIC) and the Marigold Foundation.
View article: Insulin Signaling as a Key Moderator in Myotonic Dystrophy Type 1
Insulin Signaling as a Key Moderator in Myotonic Dystrophy Type 1 Open
Myotonic dystrophy type 1 (DM1) is an autosomal dominant genetic disease characterized by multi-system involvement. Affected organ system includes skeletal muscle, heart, gastro-intestinal system and the brain. In this review, we evaluate …
View article: Genetic determinants of disease severity in the myotonic dystrophy type 1 OPTIMISTIC cohort
Genetic determinants of disease severity in the myotonic dystrophy type 1 OPTIMISTIC cohort Open
Careful characterization of the DMPK CTG repeat to define progenitor allele length and presence of variant repeats has increased utility in understanding clinical variability in a trial cohort and provides a genetic route for defini…
View article: Affective symptoms and apathy in myotonic dystrophy type 1 a systematic review and meta-analysis
Affective symptoms and apathy in myotonic dystrophy type 1 a systematic review and meta-analysis Open
View article: Structural white matter networks in myotonic dystrophy type 1
Structural white matter networks in myotonic dystrophy type 1 Open
View article: MPZL2, Encoding the Epithelial Junctional Protein Myelin Protein Zero-like 2, Is Essential for Hearing in Man and Mouse
MPZL2, Encoding the Epithelial Junctional Protein Myelin Protein Zero-like 2, Is Essential for Hearing in Man and Mouse Open
View article: Cognitive behavioural therapy with optional graded exercise therapy in patients with severe fatigue with myotonic dystrophy type 1: a multicentre, single-blind, randomised trial
Cognitive behavioural therapy with optional graded exercise therapy in patients with severe fatigue with myotonic dystrophy type 1: a multicentre, single-blind, randomised trial Open
View article: Early onset facioscapulohumeral dystrophy – a systematic review using individual patient data
Early onset facioscapulohumeral dystrophy – a systematic review using individual patient data Open