Impact of long-term elosulfase alfa on activities of daily living in patients with Morquio A syndrome in an open-label, multi-center, phase 3 extension study.

Background: Long-term safety and efficacy of elosulfase alfa enzyme replacement therapy (ERT) were assessed in 173 patients with Morquio A syndrome (mucopolysaccharidosis IVA) in a 96-week, open-label, multi-center, phase 3 extension study (MOR-005) of the pivotal 24-week, placebo-controlled study (MOR-004). Changes in efficacy endpoints were evaluated over 120weeks, from MOR-004 baseline to MOR-005 week 96. We report the impact of ERT on activities of daily living (ADL) across three domains (mobility, self-care, and caregiver-assistance), as assessed by the Mucopolysaccharidosis Health Assessment Questionnaire (MPS-HAQ) after 72 and 120weeks or approximately 1 and 2years.

Clinical outcomes in a subpopulation of adults with Morquio A syndrome: results from a long-term extension study of elosulfase alfa.

Background: This post hoc subanalysis examined outcomes in adult patients with Morquio A (mucopolysaccharidosis IVA) who received enzyme replacement therapy (ERT) with elosulfase alfa over a 120-weeks period. Patients ≥18 years of age evaluated in an open-label, long-term extension study of elosulfase alfa (modified per protocol [MPP], n = 32; intent-to-treat [ITT], n = 37; MOR-005; NCT01415427) were compared with the ≥18-year-old untreated population with 2-years follow-up from a Morquio A natural history study (n = 10; MorCAP; NCT00787995). The MOR-005 MPP population excluded patients who underwent orthopedic surgical procedures or were noncompliant with study protocol (defined as missing ≥20% of ERT infusions).

The effect of galsulfase enzyme replacement therapy on the growth of patients with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome).

Mucopolysaccharidosis (MPS) VI is an autosomal recessive lysosomal storage disorder arising from deficient activity of N-acetylgalactosamine-4-sulfatase (arylsulfatase B) and subsequent intracellular accumulation of the glycosaminoglycans (GAGs) dermatan sulfate and chondroitin-4-sulfate. Manifestations are multi-systemic and include skeletal abnormalities such as dysostosis multiplex and short stature. Reference height-for-age growth charts for treatment-naïve MPS VI patients have been published for both the slowly and rapidly progressing populations.

Impact of long-term elosulfase alfa treatment on respiratory function in patients with Morquio A syndrome.

Objective: To present long-term respiratory function outcomes from an open-label, multi-center, phase 3 extension study (MOR-005) of elosulfase alfa enzyme replacement therapy (ERT) in patients with Morquio A syndrome.

Methods: In part 1 of MOR-005, patients initially randomized to ERT in the 24-week pivotal study (MOR-004) remained on their regimen (2.0 mg/kg/week or every other week); placebo patients were re-randomized to one of the two regimens.

Long-term endurance and safety of elosulfase alfa enzyme replacement therapy in patients with Morquio A syndrome.

Long-term efficacy and safety of elosulfase alfa enzyme replacement therapy were evaluated in Morquio A patients over 96weeks (reaching 120weeks in total from pre-treatment baseline) in an open-label, multi-center, phase III extension study. During this extension of a 24-week placebo-controlled phase III study, all patients initially received 2.0mg/kg elosulfase alfa either weekly or every other week, prior to establishment of 2.

Pre- and Post-Race Hydration Status in Hyponatremic and Non-Hyponatremic Ultra-Endurance Athletes.

The monitoring of body mass (BM), plasma sodium concentration ([Na⁺]) and urinary specific gravity (Usg) are commonly used to help detect and prevent over- or dehydration in endurance athletes. We investigated pre-and post-race hydration status in 113 amateur 24-h ultra-runners, 100-km ultra-runners, multi-stage mountain bikers and 24-h mountain bikers, which drank ad libitum without any intervention and compared results of hyponatremic and non-hyponatremic finishers. On average, pre-race plasma [Na⁺] and both pre- and post-race levels of Usg and BM were not significantly different between both groups.

Changes in the cortisol awakening response (CAR) following participation in mindfulness-based stress reduction in women who completed treatment for breast cancer.

Background: Changes in the cortisol awakening response (CAR) were studied in women participating in a Mindfulness-Based Stress Reduction (MBSR) program after completion of their medical treatment for breast cancer.

Method: Thirty-three women completed questionnaires pre- and post-MBSR pertaining to: stress, depressive symptomatology, and medical symptoms. The CAR was assessed on 3 days pre- and 3 days post-MBSR as a biological marker of stress.

Weathering storms: a cohort study of how participation in a mindfulness-based stress reduction program benefits women after breast cancer treatment.

Introduction: A growing number of psychosocial interventions are being offered to cancer patients during and after their medical treatment. Here, we examined whether Mindfulness-Based Stress Reduction (MBSR), a stress management course, helps women to cope better with stress and illness once their breast cancer treatment is completed. Our aim was to understand how MBSR may benefit those who participate in the course.

Cortisol as a marker for improvement in mindfulness-based stress reduction.

While much attention has been devoted to examining the beneficial effects of Mindfulness-Based Stress Reduction programs on patients' ability to cope with various chronic medical conditions, most studies have relied on self-report measures of improvement. Given that these measures may not accurately reflect physiological conditions, there is a need for an objective marker of improvement in research evaluating the beneficial effects of stress management programs. Cortisol is the major stress hormone in the human organism and as such is a promising candidate measure in the study of the effects of Mindfulness-Based Stress Reduction programs.

Sex steroid hormones and cognitive functioning in healthy, older men.

The precise impact of age-related changes in hormone levels on cognition in men is still unclear due to differing study designs and contradictory findings. This study was undertaken to examine the relationship between endogenous sex hormone levels and cognitive functioning in healthy older men using a comprehensive battery of neuropsychological tests and measurement of serum sex hormone levels. Verbal learning and memory, visual-motor processing, spatial abilities, working memory and attention, and levels of testosterone and estradiol were evaluated in 54 healthy older men.

A randomized controlled trial of add-back estrogen or placebo on cognition in men with prostate cancer receiving an antiandrogen and a gonadotropin-releasing hormone analog.

The effects of testosterone (T) and estradiol (E(2)) on cognition in men are confounded in extant studies. This randomized, placebo-controlled trial was undertaken to investigate the possible effects of E(2) on cognition in older men. Twenty-five men with prostate cancer (mean age: 71.

Growth and nutritional indexes in early life predict pulmonary function in cystic fibrosis.

Objective: To determine the relation of growth and nutritional status to pulmonary function in young children with cystic fibrosis (CF).

Study Design: The relation of weight-for-age (WFA), height-for-age (HFA), percent ideal body weight (%IBW), and signs of lung disease at age 3 years with pulmonary function at age 6 years was assessed in 931 patients with CF. Associations of changes in WFA from age 3 to 6 on pulmonary function were also assessed.