A natural history study to track brain and spinal cord changes in individuals with Friedreich's ataxia: TRACK-FA study protocol.

Introduction: Drug development for neurodegenerative diseases such as Friedreich's ataxia (FRDA) is limited by a lack of validated, sensitive biomarkers of pharmacodynamic response in affected tissue and disease progression. Studies employing neuroimaging measures to track FRDA have thus far been limited by their small sample sizes and limited follow up. TRACK-FA, a longitudinal, multi-site, and multi-modal neuroimaging natural history study, aims to address these shortcomings by enabling better understanding of underlying pathology and identifying sensitive, clinical trial ready, neuroimaging biomarkers for FRDA.

Hospital utilization rates following antipsychotic dose reduction in mood disorders: implications for treatment of tardive dyskinesia.

Background: The relative benefits and risks of long-term maintenance treatment with antipsychotics have not been well studied in patients with bipolar disorder and major depressive disorder. For example, while antipsychotic dose reduction has been recommended in the management of serious side effects associated with antipsychotics, there is limited evidence on the impact of lowering doses on the course of underlying mood disorders.

Methods: This retrospective cohort study analyzed the impact of antipsychotic dose reduction in patients with bipolar disorder or major depressive disorder.

Ataluren use in patients with nonsense mutation Duchenne muscular dystrophy: patient demographics and characteristics from the STRIDE Registry.

Strategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, multicenter registry providing real-world evidence regarding ataluren use in patients with nonsense mutation Duchenne muscular dystrophy (DMD) in clinical practice (NCT02369731). Here, we describe the initial demographic characteristics of the registry population. Patients will be followed up from enrollment for ≥5 years or until study withdrawal.

The burden of tardive dyskinesia secondary to antipsychotic medication use among patients with mental disorders.

To assess the impact of developing tardive dyskinesia (TD), both with and without other pre-existing extrapyramidal symptoms (EPS), on healthcare resource utilization (HRU) among patients with mental disorders receiving antipsychotic medications. Data on patients receiving antipsychotics who had schizophrenia, major depressive disorder or bipolar disorder were extracted from a Medicaid claims database. Separate cohorts of TD patients with and without other EPS ("TD + EPS" and "TD non-EPS") were constructed and matched to patients in a non-TD/EPS control cohort at a ∼1:5 ratio.

Hospital utilization rates following antipsychotic dose reductions: implications for tardive dyskinesia.

Background: Data are limited on the benefits and risks of dose reduction in managing side effects associated with antipsychotic treatment. As an example, antipsychotic dose reduction has been recommended in the management of tardive dyskinesia (TD), yet the benefits of lowering doses are not well studied. However, stable maintenance treatment is essential to prevent deterioration and relapse in schizophrenia.