Nikoletta Nikolenko
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View article: Periostin as a blood biomarker of muscle cell fibrosis, cardiomyopathy and disease severity in myotonic dystrophy type 1
Periostin as a blood biomarker of muscle cell fibrosis, cardiomyopathy and disease severity in myotonic dystrophy type 1 Open
View article: Circulating small RNA signatures differentiate accurately the subtypes of muscular dystrophies: small-RNA next-generation sequencing analytics and functional insights
Circulating small RNA signatures differentiate accurately the subtypes of muscular dystrophies: small-RNA next-generation sequencing analytics and functional insights Open
Muscular dystrophies are a group of rare and severe inherited disorders mainly affecting the muscle tissue. Duchene Muscular Dystrophy, Myotonic Dystrophy types 1 and 2, Limb Girdle Muscular Dystrophy and Facioscapulohumeral Muscular Dystr…
View article: Circulating small RNA signatures differentiate accurately the subtypes of muscular dystrophies: small-RNA next-generation sequencing analytics and functional insights
Circulating small RNA signatures differentiate accurately the subtypes of muscular dystrophies: small-RNA next-generation sequencing analytics and functional insights Open
Muscular dystrophies are a group of rare and severe inherited disorders mainly affecting the muscle tissue. Duchene Muscular Dystrophy, Myotonic Dystrophy types 1 and 2, Limb Girdle Muscular Dystrophy and Facioscapulohumeral Muscular Dystr…
View article: miR-223-3p and miR-24-3p as novel serum-based biomarkers for myotonic dystrophy type 1
miR-223-3p and miR-24-3p as novel serum-based biomarkers for myotonic dystrophy type 1 Open
View article: Dysregulation of GSK3β-Target Proteins in Skin Fibroblasts of Myotonic Dystrophy Type 1 (DM1) Patients
Dysregulation of GSK3β-Target Proteins in Skin Fibroblasts of Myotonic Dystrophy Type 1 (DM1) Patients Open
Myotonic dystrophy type 1 (DM1) is the most common monogenetic muscular disorder of adulthood. This multisystemic disease is caused by CTG repeat expansion in the 3 ′ -untranslated region of the DM1 protein kinase gene called DMPK. DMPK en…
View article: A Phase 2 Study of AMO-02 (Tideglusib) in Congenital and Childhood-Onset Myotonic Dystrophy Type 1 (DM1)
A Phase 2 Study of AMO-02 (Tideglusib) in Congenital and Childhood-Onset Myotonic Dystrophy Type 1 (DM1) Open
AMO-02 has favorable pharmacokinetic and clinical risk/benefit profiles meriting further study as a potential treatment for congenital and childhood-onset type 1 myotonic dystrophy.
View article: Change over time in ability to perform activities of daily living in myotonic dystrophy type 1
Change over time in ability to perform activities of daily living in myotonic dystrophy type 1 Open
Objective The objective of this longitudinal, observational study was to investigate change over time in ability to perform activities of daily living in myotonic dystrophy type 1 (DM1). Methods Adults with genetically confirmed DM1 were r…
View article: Activities of daily living in myotonic dystrophy type 1
Activities of daily living in myotonic dystrophy type 1 Open
We show that DM1 impairs performance of activities of daily living, in particular those requiring a high degree of muscle strength, stability and coordination. Yet, across the evolution of the disease, the majority of patients will still b…
View article: Design, Conduct, and Use of Patient Preference Studies in the Medical Product Life Cycle: A Multi-Method Study
Design, Conduct, and Use of Patient Preference Studies in the Medical Product Life Cycle: A Multi-Method Study Open
Objectives: To investigate stakeholder perspectives on how patient preference studies (PPS) should be designed and conducted to allow for inclusion of patient preferences in decision-making along the medical product life cycle (MPLC…
View article: Design, Conduct and Use of Patient Preference Studies in the Medical Product Life Cycle
Design, Conduct and Use of Patient Preference Studies in the Medical Product Life Cycle Open
Objectives: To investigate stakeholder perspectives on how patient preference studies
\n(PPS) should be designed and conducted to allow for inclusion of patient preferences in
\ndecision-making along the medical product life cycle (MPLC), …
View article: Reproductive Cancer Risk Factors in Women With Myotonic Dystrophy (DM): Survey Data From the US and UK DM Registries
Reproductive Cancer Risk Factors in Women With Myotonic Dystrophy (DM): Survey Data From the US and UK DM Registries Open
Introduction: Recent evidence demonstrates that women with myotonic dystrophy type 1 are at increased risk of reproductive organ tumors. However, studies of reproductive cancer risk factors in those patients are lacking. Methods:…
View article: Analysis of the functional capacity outcome measures for myotonic dystrophy
Analysis of the functional capacity outcome measures for myotonic dystrophy Open
Objectives Defining clinically relevant outcome measures for myotonic dystrophy type 1 (DM1) that can be valid and feasible for different phenotypes has proven problematic. The Outcome Measures for Myotonic Dystrophy (OMMYD) group proposed…
View article: Patient Preferences in the Medical Product Life Cycle: What do Stakeholders Think? Semi-Structured Qualitative Interviews in Europe and the USA
Patient Preferences in the Medical Product Life Cycle: What do Stakeholders Think? Semi-Structured Qualitative Interviews in Europe and the USA Open
View article: Disease burden of myotonic dystrophy type 1
Disease burden of myotonic dystrophy type 1 Open
View article: Survival patterns and cancer determinants in families with myotonic dystrophy type 1
Survival patterns and cancer determinants in families with myotonic dystrophy type 1 Open
Background and purpose Research indicates that patients with myotonic dystrophy type 1 ( DM 1) are at increased risk of cancer and early death. Family data may provide insights given DM 1 phenotypic heterogeneity, the broad range of non‐mu…
View article: Analyzing walking speeds with ankle and wrist worn accelerometers in a cohort with myotonic dystrophy
Analyzing walking speeds with ankle and wrist worn accelerometers in a cohort with myotonic dystrophy Open
Background: Accelerometers are accurate tools to assess movement and physical activity. However, interpreting standardly used outputs is not straightforward for populations with impaired mobility.Methods: The applicability of…
View article: GNE myopathy: from clinics and genetics to pathology and research strategies
GNE myopathy: from clinics and genetics to pathology and research strategies Open
View article: Risk of skin cancer among patients with myotonic dystrophy type 1 based on primary care physician data from the <scp>U</scp>.<scp>K</scp>. <scp>C</scp>linical <scp>P</scp>ractice <scp>R</scp>esearch <scp>D</scp>atalink
Risk of skin cancer among patients with myotonic dystrophy type 1 based on primary care physician data from the <span>U</span>.<span>K</span>. <span>C</span>linical <span>P</span>ractice <span>R</span>esearch <span>D</span>atalink Open
Myotonic dystrophy type 1 (DM1) is an inherited multisystem neuromuscular disorder caused by a CTG trinucleotide repeat expansion in the DMPK gene. Recent evidence documents that DM1 patients have an increased risk of certain cancers, but …
View article: Benign and malignant tumors in the UK myotonic dystrophy patient registry
Benign and malignant tumors in the UK myotonic dystrophy patient registry Open
Introduction In light of recent evidence indicating that cancer is part of the myotonic dystrophy (DM) phenotype, we assessed the prevalence of benign and malignant tumors among 220 patients enrolled in the UK Myotonic Dystrophy Patient Re…
View article: The UK Myotonic Dystrophy Patient Registry: facilitating and accelerating clinical research
The UK Myotonic Dystrophy Patient Registry: facilitating and accelerating clinical research Open
View article: Functional impairment in patients with myotonic dystrophy type 1 can be assessed by an ataxia rating scale (SARA)
Functional impairment in patients with myotonic dystrophy type 1 can be assessed by an ataxia rating scale (SARA) Open
View article: Impact of subarachnoid haemorrhage Canadian clinical decision rules for investigation of acute headache: a retrospective case note review
Impact of subarachnoid haemorrhage Canadian clinical decision rules for investigation of acute headache: a retrospective case note review Open
View article: Long-term efficiency of training program for patients with artificial heart valves to improve theadherence to treatment and quality of life
Long-term efficiency of training program for patients with artificial heart valves to improve theadherence to treatment and quality of life Open