Prospective observational study of FKRP-related limb-girdle muscular dystrophy R9: A GRASP consortium study.

Objective: Limb-girdle muscular dystrophy R9 (LGMDR9, formerly known as LGMD2I), caused by variants in the fukutin-related protein (FKRP) gene leads to progressive muscle weakness of the shoulder and pelvic limb-girdles and loss of motor function over time. Clinical management and future trial design are improved by determining which standardized clinical outcome assessments (COA) of function are most appropriate to capture disease presentation and progression, informing endpoint selection and enrollment criteria. The purpose of our study was to evaluate the cross-sectional validity and reliability of clinical outcome assessments in patients with FKRP-related LGMDR9 participating in the Genetic Resolution and Assessments Solving Phenotypes in LGMD (GRASP) natural history study.

Public sector replacement of privately funded pharmaceutical R&D: cost and efficiency considerations.

Aims: Economic studies have found that public support of basic medical research provides important long-term benefits. In response to suggestions that private pharmaceutical research and development (R&D) funding could be totally replaced by public funding, we investigate the economic implications of such a substitution in funding roles that maintain the recent pace of pharmaceutical innovation.

Materials And Methods: Total lifecycle R&D costs were estimated using the latest available R&D expenditures per novel molecule entering clinical trials, likelihood of approval, pre-clinical and post-approval expenditures, using a published survey and a review of publicly available financial accounts from US-listed multinational developers.

Understanding the impact of slowing disease progression for individuals with biomarker-confirmed early symptomatic Alzheimer's disease.

Recent advances in development of amyloid-targeting therapies support the potential to slow the rate of progression of Alzheimer's disease. We conducted a narrative review of published evidence identified through a targeted search of the MEDLINE and EMBASE databases (2020-2023), recent presentations at disease-specific conferences, and data updates from cohort studies in Alzheimer's disease to describe the trajectory of the progression of Alzheimer's disease. Our findings enable the interpretation of clinical trial results and the value associated with slowing disease progression across outcomes of relevance to patients, care partners, clinicians, researchers and policymakers.

Real-World Incidence of Adverse Clinical Outcomes Among People With Coronary Artery Disease and/or Peripheral Artery Disease in Relation to Vascular Risk in the United States.

Presence of polyvascular disease, diabetes, heart failure, or renal insufficiency in patients with chronic coronary artery disease (CAD) and peripheral artery disease (PAD) are associated with increased risks of adverse events, including major adverse cardiovascular events (MACEs) and major adverse limb events (MALEs). In this retrospective observational study using administrative claims data from Optum's deidentified Clinformatics Data Mart Database from January 2016 to September 2021, we described the incidence rates of MACEs, MALEs, and major thrombotic vascular events in patients with CAD or PAD stratified by the presence of risk factors (i.e.

Unintended Consequences of Increased Out-of-Pocket Costs During Medicare Coverage Gap on Anticoagulant Discontinuation and Stroke.

Introduction: This study aims to assess the risk of direct oral anticoagulant (DOAC) discontinuation among Medicare beneficiaries with non-valvular atrial fibrillation (NVAF) who reach the Medicare coverage gap stratified by low-income subsidy (LIS) status and the impact of DOAC discontinuation on rates of stroke and systemic embolism (SE) among beneficiaries with increased out-of-pocket (OOP) costs due to not receiving LIS.

Methods: In this retrospective cohort study, Medicare claims data (2015-2020) were used to identify beneficiaries with NVAF who initiated rivaroxaban or apixaban and entered the coverage gap during ≥ 1 year. DOAC discontinuation rates during the coverage gap were stratified by receipt of Medicare Part D Low-Income Subsidy (LIS), a proxy for not experiencing increased OOP costs.

How I Treat Chronic Inflammatory Demyelinating Polyneuropathy Podcast.

Chronic inflammatory demyelinating polyneuropathy (CIDP) is an acquired immune-mediated neuropathy that typically presents with progressive or relapsing, symmetric, proximal, and distal weakness of upper and lower limbs, sensory involvement of at least two limbs, and decreased or absent deep tendon reflexes. The symptoms of CIDP can be similar to those of other neuropathies, making diagnosis difficult, which can often lead to delays in correct diagnosis and treatment. The updated European Academy of Neurology/Peripheral Nerve Society (EAN/PNS) 2021 guideline outlines a set of diagnostic criteria that help to identify CIDP with high accuracy and provides recommendations for the treatment of CIDP.

'Didn't See the Need': Misperceptions about glucagon from the perspectives of people with diabetes and their caregivers.

Aims: Severe hypoglycaemia among people with diabetes who use insulin can be a life-threatening complication if left untreated. Although glucagon has been approved for treatment of hypoglycaemia since the 1960s, it has been underutilized. We aimed to understand the perceptions of people with diabetes and their caregivers about glucagon.

Real-World Racial Variation in Treatment and Outcomes Among Patients with Peripheral Artery Disease.

Introduction: Prior studies have found considerable disparities in prevalence and outcomes for patients with peripheral arterial disease (PAD). This study compared rates of diagnostic testing, treatment patterns, and outcomes after diagnosis of PAD among commercially insured Black and White patients in the United States.

Methods: Optum's de-identified Clinformatics Data Mart Database (1/2016-6/2021) were used to identify Black and White patients with PAD; first PAD diagnosis was deemed study index date.

Disease Progression and Longitudinal Clinical Outcomes of Lewy Body Dementia in the NACC Database.

Introduction: As the identification of Lewy body dementia (LBD) is often confirmed postmortem, there is a paucity of evidence on the progression of disease antemortem. This study aimed to comprehensively assess the course of LBD over time across cognitive, functional, and neuropsychiatric outcomes using real-world data.

Methods: Adults with at least one visit to an Alzheimer's Disease Center with a diagnosis of mild cognitive impairment/dementia (index date), indication of LBD, and at least one follow-up visit were identified in the National Alzheimer's Coordinating Center database (September 2005-June 2020).

Reasons for treatment changes in adults with attention-deficit/hyperactivity disorder: a chart review study.

Background: Adults with attention-deficit hyperactivity disorder (ADHD) often cycle through multiple treatments for reasons that are not well documented. This study analyzed the reasons underlying treatment changes among adults treated for ADHD in a real-world setting.

Methods: Data were collected via an online reporting form completed by eligible physicians between October and November 2020.

Randomized Phase 2 Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease.

Background And Objectives: The goal of this work was to determine whether locally acting ACE-083 is safe and well tolerated and increases muscle volume, motor function, and quality of life (QoL) in adults with Charcot-Marie-Tooth disease (CMT) type 1.

Methods: This phase 2 study enrolled adults with CMT1 or CMTX (N = 63). Part 1 was open label and evaluated the safety and tolerability of different dose levels of ACE-083 for use in part 2.

Epidemiology and economic burden of Lewy body dementia in the United States.

Objective: To describe the trends in epidemiology, healthcare resource use (HCRU), and costs associated with Lewy body dementia (LBD), dementia with Lewy bodies (DLB), and Parkinson's disease dementia (PDD) in the United States.

Methods: This retrospective study used administrative claims data for Medicare fee-for-service (2010-2018) and commercially-insured beneficiaries (2010-2017). The annual prevalence and incidence were calculated among the Medicare beneficiaries by dividing the number of prevalent or incident LBD, DLB, and PDD patients by the total eligible population of that calendar year.

Randomized phase 2 study of ACE-083, a muscle-promoting agent, in facioscapulohumeral muscular dystrophy.

Introduction/aims: Facioscapulohumeral muscular dystrophy (FSHD) is a slowly progressive muscular dystrophy without approved therapies. In this study we evaluated whether locally acting ACE-083 could safely increase muscle volume and improve functional outcomes in adults with FSHD.

Methods: Participants were at least 18 years old and had FSHD1/FSHD2.

Toward a more comprehensive understanding of the emotional side of type 2 diabetes: A re-envisioning of the assessment of diabetes distress.

Aims: To develop an updated, more theoretically sound system for describing and assessing diabetes-related emotional distress (DD) in adults with type 2 diabetes (T2D).

Methods: Items were developed from qualitative interviews with 11 adults with T2D and 6 clinicians, then categorized as reflecting a core emotional experience of DD or a primary source of DD. Items were then administered to a national sample of TCOYD Registry participants.

The experience of a severe hypoglycaemic event from the perspective of people with diabetes and their caregivers: "What am I going to do?".

Aims: Among people with diabetes using insulin, severe hypoglycaemia (SH) can be a life-threatening complication, if untreated. The personal experiences during an SH event from the perspectives of people with diabetes and their caregivers are not well-characterized. This study assessed the perceptions of the event and the decision making processes of people with diabetes (T1D n = 36; T2D n = 24) and their caregivers during SH events.

Classification, Prediction, and Concordance of Cognitive and Functional Progression in Patients with Mild Cognitive Impairment in the United States: A Latent Class Analysis.

Background: Progression trajectories of patients with mild cognitive impairment (MCI) are currently not well understood.

Objective: To classify patients with incident MCI into different latent classes of progression and identify predictors of progression class.

Methods: Participants with incident MCI were identified from the US National Alzheimer's Coordinating Center Uniform Data Set (09/2005-02/2019).

Incidence of Major Atherothrombotic Vascular Events among Patients with Peripheral Artery Disease after Revascularization.

Background: Patients with peripheral artery disease (PAD) treated with lower extremity revascularization are at increased risk of major atherothrombotic vascular events (acute limb ischemia (ALI), major non-traumatic lower-limb amputation, myocardial infarction (MI), ischemic stroke, and cardiovascular (CV)-related death). This study assessed the incidence of major atherothrombotic vascular events, venous thromboembolism (VTE) events and rates of subsequent lower extremity revascularizations in the real-world among patients with PAD after revascularization.

Methods: Patients aged ≥50 years with PAD who underwent peripheral revascularization were identified from Optum Clinformatics Data Mart claims database (Q1/2014-Q2/2019).

Overcoming psychological insulin resistance: A practical guide for healthcare professionals.

Despite the demonstrated benefits of using insulin, nearly a third of the patients with type 2 diabetes (T2D) are initially reluctant to initiate insulin therapy when it is first recommended by their healthcare provider (HCP). Several studies have documented the reasons for this phenomenon known as psychological insulin resistance (PIR) and also identified actionable strategies for HCPs to assist people with T2D to overcome their PIR. However, most strategies are based on the experiences of HCPs, rather than of patients.

Healthcare resource utilization and costs of major atherothrombotic vascular events among patients with peripheral artery disease after revascularization.

Aims: Peripheral artery disease (PAD), often treated with lower extremity revascularization, is associated with risk of major atherothrombotic vascular events (acute limb ischemia [ALI], major non-traumatic lower-limb amputation, myocardial infarction [MI], ischemic stroke, cardiovascular death). This study aims to assess healthcare resource utilization and costs of such events among patients with PAD after revascularization.

Materials And Methods: Patients aged ≥50 years with PAD who were treated with lower-extremity revascularization were identified from Optum Clinformatics Data Mart claims database (01/2014-06/2019).

Cost-per-remitter with esketamine nasal spray versus standard of care for treatment-resistant depression.

Estimate the cost-per-remitter with esketamine nasal spray plus an oral antidepressant (ESK + oral AD) versus oral AD plus nasal placebo (oral AD + PBO) among patients with treatment-resistant depression. An Excel-based model was developed to estimate the cost-per-remitter for ESK + oral AD versus oral AD + PBO over 52 weeks from multiple US payer perspectives. Clinical end points and cost inputs were derived from clinical trials and the literature, respectively.

Measuring The Impact Of Air Pollution On Health Care Costs.

Air pollution contributes to the development of numerous adverse human health outcomes. The Environmental Protection Agency's Environmental Benefits Mapping and Analysis Program-Community Edition (BenMAP-CE) tool is widely used in estimating the health care costs of air pollution and in the development of federal and state regulations and policy. Its default features consider only the costs of hospital and emergency department admissions.

Disease severity at the time of initial cognitive assessment is related to prior health-care resource use burden.

Introduction: Research has shown increased health-care resource use (HRU) among patients with Alzheimer's disease and related disorders (ADRD) well before diagnosis, but the degree to which HRU is correlated with disease severity at the time of initial assessment is not well documented.

Methods: Retrospective analysis of linked medical records and claims data for three cohorts: mild ADRD (first [index] Mini-Mental State Examination [MMSE] ≥20), moderate/severe ADRD (index MMSE < 20), controls without cognitive impairment. HRU during the pre-index year was compared using multivariate regressions.

Key Strategies for Overcoming Psychological Insulin Resistance in Adults with Type 2 Diabetes: The UK Subgroup in the EMOTION Study.

Introduction: Many patients with type 2 diabetes mellitus (T2DM) delay initiation of insulin therapy despite healthcare professional (HCP) advice. This phenomenon has been referred to as 'psychological insulin resistance' (PIR), and various contributing factors have been identified. Studies discussing approaches to overcoming PIR are lacking.