Project description:Peripheral neuropathy is a common neurologic disorder, affecting 2% to 8% of the population in population-based studies with confirmation by neurologist examination. These prevalence numbers are remarkably stable across developed countries. In 1999, 8.6% of Medicare beneficiaries had neuropathy as a primary or secondary diagnosis, and the cost of treatment was estimated at $3.5 billion (Consumer Price Index adjusted to 2013 $4.9 billion), which did not include outpatient medications. Peripheral neuropathy has many causes and varies in regard to its clinical manifestations and severity. Distal symmetric polyneuropathy (DSP) is the most common pattern of peripheral neuropathy generally and the most common phenotype of neuropathy due to diabetes. Reported prevalence rates of DSP among diabetic patients range from 15% to 37% across large population-based studies, and the prevalence among those with impaired glucose tolerance has been reported to be 11%. DSP can result in weakness, sensory loss, pain, autonomic dysfunction, gait impairment, falls, disability, and impaired quality of life.

| S-EPMC4032209 | biostudies-literature

Quality improvement in neurology: Multiple sclerosis quality measures: Executive summary.

Project description: Not available

| S-EPMC4662704 | biostudies-literature

Quality improvement in neurology: amyotrophic lateral sclerosis quality measures: report of the quality measurement and reporting subcommittee of the American Academy of Neurology.

Project description:Amyotrophic lateral sclerosis (ALS) is a lethal, progressive neurodegenerative disease characterized by loss of motor neurons.(1) Patients with ALS lose function in the limbs, speech, swallowing, and breathing muscles. The cause of the disease is still not known for most patients. Approximately 25,000 people in the United States have ALS, and 5,000 people are diagnosed with ALS annually in the United States.(1) Most patients die from respiratory failure 2 to 5 years after onset of symptoms. Cognitive dysfunction is seen in 20% to 50% of patients.(2) The disease burden for patients and caregivers is enormous. The average cost of care has been estimated at $50,000 per patient per year.(3.)

| S-EPMC3863352 | biostudies-literature

Quality improvement in neurology: AAN Parkinson disease quality measures: report of the Quality Measurement and Reporting Subcommittee of the American Academy of Neurology.

Project description:BackgroundMeasuring the quality of health care is a fundamental step toward improving health care and is increasingly used in pay-for-performance initiatives and maintenance of certification requirements. Measure development to date has focused on primary care and common conditions such as diabetes; thus, the number of measures that apply to neurologic care is limited. The American Academy of Neurology (AAN) identified the need for neurologists to develop measures of neurologic care and to establish a process to accomplish this.ObjectiveTo adapt and test the feasibility of a process for independent development by the AAN of measures for neurologic conditions for national measurement programs.MethodsA process that has been used nationally for measure development was adapted for use by the AAN. Topics for measure development are chosen based upon national priorities, available evidence base from a systematic literature search, gaps in care, and the potential impact for quality improvement. A panel composed of subject matter and measure development methodology experts oversees the development of the measures. Recommendation statements and their corresponding level of evidence are reviewed and considered for development into draft candidate measures. The candidate measures are refined by the expert panel during a 30-day public comment period and by review by the American Medical Association for Current Procedural Terminology (CPT) II codes. All final AAN measures are approved by the AAN Board of Directors.ResultsParkinson disease (PD) was chosen for measure development. A review of the medical literature identified 258 relevant recommendation statements. A 28-member panel approved 10 quality measures for PD that included full specifications and CPT II codes.ConclusionThe AAN has adapted a measure development process that is suitable for national measurement programs and has demonstrated its capability to independently develop quality measures.

| S-EPMC3014235 | biostudies-literature

Quality Improvement in Neurology: Headache Quality Measurement Set.

Project description: Not available

| S-EPMC8092124 | biostudies-literature

Quality improvement in neurology: Epilepsy Update Quality Measurement Set.

Project description: Not available

| S-EPMC4395888 | biostudies-literature

Quality improvement in neurology: Neuro-Oncology Quality Measurement Set.

Project description: Not available

| S-EPMC5909638 | biostudies-literature

Outcome Measures in Facioscapulohumeral Muscular Dystrophy Clinical Trials.

Project description:Facioscapulohumeral muscular dystrophy (FSHD) is a debilitating muscular dystrophy with a variable age of onset, severity, and progression. While there is still no cure for this disease, progress towards FSHD therapies has accelerated since the underlying mechanism of epigenetic derepression of the double homeobox 4 (DUX4) gene leading to skeletal muscle toxicity was identified. This has facilitated the rapid development of novel therapies to target DUX4 expression and downstream dysregulation that cause muscle degeneration. These discoveries and pre-clinical translational studies have opened new avenues for therapies that await evaluation in clinical trials. As the field anticipates more FSHD trials, the need has grown for more reliable and quantifiable outcome measures of muscle function, both for early phase and phase II and III trials. Advanced tools that facilitate longitudinal clinical assessment will greatly improve the potential of trials to identify therapeutics that successfully ameliorate disease progression or permit muscle functional recovery. Here, we discuss current and emerging FSHD outcome measures and the challenges that investigators may experience in applying such measures to FSHD clinical trial design and implementation.

| S-EPMC8870318 | biostudies-literature

Improvement of Duchenne muscular dystrophy phenotype following obestatin treatment.

Project description:BackgroundThis study was performed to test the therapeutic potential of obestatin, an autocrine anabolic factor regulating skeletal muscle repair, to ameliorate the Duchenne muscular dystrophy (DMD) phenotype.Methods and resultsUsing a multidisciplinary approach, we characterized the ageing-related preproghrelin/GPR39 expression patterns in tibialis anterior (TA) muscles of 4-, 8-, and 18-week-old mdx mice (n = 3/group) and established the effects of obestatin administration at this level in 8-week-old mdx mice (n = 5/group). The findings were extended to in vitro effects on human immortalized DMD myotubes. An analysis of TAs revealed an age-related loss of preproghrelin expression, as precursor of obestatin, in mdx mice. Administration of obestatin resulted in a significant increase in tetanic specific force (33.0% ± 1.5%, P < 0.05), compared with control mdx mice. Obestatin-treated TAs were characterized by reduction of fibres with centrally located nuclei (10.0% ± 1.2%, P < 0.05) together with an increase in the number of type I fibres (25.2% ± 1.7%, P < 0.05) associated to histone deacetylases/myocyte enhancer factor-2 and peroxisome proliferator-activated receptor-gamma coactivator 1? axis, and down-regulation of ubiquitin E3-ligases by inactivation of FoxO1/4, indexes of muscle atrophy. Obestatin reduced the level of contractile damage and tissue fibrosis. These observations correlated with decline in serum creatine kinase (58.8 ± 15.2, P < 0.05). Obestatin led to stabilization of the sarcolemma by up-regulation of utrophin, ?-syntrophin, ?-dystroglycan, and ?7?1-integrin proteins. These pathways were also operative in human DMD myotubes.ConclusionsThese results highlight the potential of obestatin as a peptide therapeutic for preserving muscle integrity in DMD, thus allowing a better efficiency of gene or cell therapy in a combined therapeutic approach.

| S-EPMC6240759 | biostudies-literature

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