Risdiplam for the Use of Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is one of the leading causes of death in infants related to the degeneration of neurons. Currently, there are no curative treatment options for SMA, and many options available may not be feasible. This review presents the background, clinical studies, and indications for the use of Risdiplam in treating SMA. SMA causes a decrease in the production of survival motor neuron proteins (SMN) and current treatments target to increase the expression of SMN. Risdiplam is the first and only oral medication to be approved to treat SMA. As an SMN2 splicing modifier, it has provided stronger systemic therapies than previous intrathecal and gene replacement therapies. There have been many efforts to treat SMA with multidisciplinary approaches. These include intrathecal injections to gene replacement therapies. However, these have been faced with limitations such as reaching a good therapeutic dose in systemic tissues, route of administration, and price. Risdiplam is currently the only orally administered drug approved by the FDA for the treatment of SMA. It not only provides a good therapeutic window to systemic tissues but allows for a non-invasive approach in infants. Further investigation and comparison on the safety profile of Risdiplam due to its broader systemic effect should be considered with other available therapies

Implications of Trauma among Male and Female Offenders

Criminal behaviour is believed to arise from a multiplicity of factors, including unemployment and poverty [1,2], low self-control [3], psychological issues [4,5], early conduct problems [6], childhood physical and sexual abuse disorder [5], and social bonding in child- and adulthood [7]. Social-structural influences like family conflict/disruption, financial resources, child-parent and school/peer attachment and abuse and neglect in childhood have lasting impressions, leading to multiple problems including delinquency and later criminal activity, substance use/abuse, mental illness and poor self-rated health [8-12]. The consequences of such behaviour include financial losses, injury, and death that together have significant personal and societal costs. Society also bears the burden of incarcerating and rehabilitating offenders; a burden that is not trivial. Direct costs of imprisonment in Canada approach $3.5 billion annually; in the US the cost is substantially higher, approaching$74 billion [13]. [...

Predictors and outcomes of patient safety culture in hospitals

<p>Abstract</p> <p>Background</p> <p>Developing a patient safety culture was one of the recommendations made by the Institute of Medicine to assist hospitals in improving patient safety. In recent years, a multitude of evidence, mostly originating from developed countries, has been published on patient safety culture. One of the first efforts to assess the culture of safety in the Eastern Mediterranean Region was by El-Jardali et al. (2010) in Lebanon. The study entitled "The Current State of Patient Safety Culture: a study at baseline" assessed the culture of safety in Lebanese hospitals. Based on study findings, the objective of this paper is to explore the association between patient safety culture predictors and outcomes, taking into consideration respondent and hospital characteristics. In addition, it will examine the correlation between patient safety culture composites.</p> <p>Methods</p> <p>Sixty-eight hospitals and 6,807 respondents participated in the study. The study which adopted a cross sectional research design utilized an Arabic-translated version of the Hospital Survey on Patient Safety Culture (HSOPSC). The HSOPSC measures 12 patient safety composites. Two of the composites, in addition to a patient safety grade and the number of events reported, represented the four outcome variables. Bivariate and mixed model regression analyses were used to examine the association between the patient safety culture predictors and outcomes.</p> <p>Results</p> <p>Significant correlations were observed among all patient safety culture composites but with differences in the strength of the correlation. Generalized Estimating Equations for the patient safety composite scores and respondent and hospital characteristics against the patient safety grade and the number of events reported revealed significant correlations. Significant correlations were also observed by linear mixed models of the same variables against the frequency of events reported and the overall perception of safety.</p> <p>Conclusion</p> <p>Event reporting, communication, patient safety leadership and management, staffing, and accreditation were identified as major patient safety culture predictors. Investing in practices that tackle these issues and prioritizing patient safety is essential in Lebanese hospitals in order to improve patient safety. In addition, further research is needed to understand the association between patient safety culture and clinical outcomes.</p

Fatal alcohol intoxication in women: A forensic autopsy study from Slovakia

<p>Abstract</p> <p>Background</p> <p>Plenty of information related to alcoholism can be found in the literature, however, the studies have mostly dealt with the predominance of male alcoholism and data related to addiction in women are desperately scarce and difficult to find. Basic demographic data focusing on the impact of acute alcohol intoxication on the circumstances of death and social behaviour in the alcohol addicted female population are needed especially in the prevention of alcohol related mortality.</p> <p>Methods</p> <p>A retrospective forensic autopsy study of all accidental deaths due to alcohol intoxication over a 12-year period was performed in order to evaluate the locations, circumstances, mechanisms and causes of death.</p> <p>Results</p> <p>A sample of 171 cases of intoxicated women who died due to blood alcohol concentration (BAC) equal to or higher than 2 g/kg was selected. Among them 36.26% (62/171) of women died due to acute alcohol intoxication (AAI). We noted an increase in the number of deaths in women due to AAI from 2 in 1994 up to 5 in 2005 (an elevation of 150% between the years 1994-2005). The age structure of deaths in women due to BAC and AAI followed the Gaussian distribution with a dominant group of women aged 41-50 years (45.16% and 35.09% respectively). The most frequent place of death (98%) among women intoxicated by alcohol was their own home. The study suggests a close connection between AAI and violence against women.</p> <p>Conclusions</p> <p>The increasing number of cases of death of women suffering from AAI has drawn attention to the serious problem of alcoholism in women in the Slovak Republic during the process of integration into "western" lifestyle and culture.</p

Pain in veterans of the Gulf War of 1991: a systematic review

BACKGROUND: Veterans of the Persian Gulf War of 1991 have reported a range of adverse health symptoms. This systematic review aims to identify all studies that have compared the prevalence of symptoms of pain in veterans of the Gulf War to that in a non-Gulf military comparison group, and to determine whether Gulf War veterans are at increased risk of reporting pain. METHODS: Studies published between January 1990 and May 2004 were identified by searching a large number of electronic databases. Reference lists and websites were also searched and key researchers were contacted. Studies were included if they reported the prevalence of any symptom or condition that included the word "pain" in Gulf War veterans and in a comparison group of non-Gulf veterans. 2401 abstracts were independently reviewed by two authors. RESULTS: Twenty studies fulfilled the inclusion criteria. Five main sites of pain were identified (muscle, joint, chest/heart, back and abdominal pain) and separate meta-analyses were performed to summarise the results related to each site. A greater proportion of Gulf veterans reported symptoms at each site of pain when compared to a non-Gulf military group. Gulf deployment was most strongly associated with abdominal pain, with Gulf veterans being more than three times more likely to report such pain than a comparison group (OR 3.23; 95%CI 2.31–4.51). Statistical heterogeneity between study estimates was significant, probably due to variation in measured periods of prevalence and symptom measurement methods. CONCLUSION: A higher proportion of veterans of the Persian Gulf War of 1991 reported symptoms of pain than military comparison groups. This is consistent with previously demonstrated increased reporting of more general symptoms (fatigue, multiple chemical sensitivity, post traumatic stress disorder) in these veterans compared with non-Gulf military groups. However, the primary studies were heterogeneous and varied greatly in quality

Reconciling disparate prevalence rates of PTSD in large samples of US male Vietnam veterans and their controls

BACKGROUND: Two large independent studies funded by the US government have assessed the impact of the Vietnam War on the prevalence of PTSD in US veterans. The National Vietnam Veterans Readjustment Study (NVVRS) estimated the current PTSD prevalence to be 15.2% while the Vietnam Experience Study (VES) estimated the prevalence to be 2.2%. We compared alternative criteria for estimating the prevalence of PTSD using the NVVRS and VES public use data sets collected more than 10 years after the United States withdrew troops from Vietnam. METHODS: We applied uniform diagnostic procedures to the male veterans from the NVVRS and VES to estimate PTSD prevalences based on varying criteria including one-month and lifetime prevalence estimates, combat and non-combat prevalence estimates, and prevalence estimates using both single and multiple indicator models. RESULTS: Using a narrow and specific set of criteria, we derived current prevalence estimates for combat-related PTSD of 2.5% and 2.9% for the VES and the NVVRS, respectively. Using a more broad and sensitive set of criteria, we derived current prevalence estimates for combat-related PTSD of 12.2% and 15.8% for the VES and NVVRS, respectively. CONCLUSION: When comparable methods were applied to available data we reconciled disparate results and estimated similar current prevalences for both narrow and broad definitions of combat-related diagnoses of PTSD

Nitrous Oxide Inhalation Among Adolescents: Prevalence, Correlates, and Co-Occurrence with Volatile Solvent Inhalation

Few studies have examined the prevalence of nitrous oxide (NO) inhalation or co-occurrence of NO and volatile solvent (VS) use in adolescents. Study aims were to (1) describe the independent and conjoint prevalence of NO and VS use in incarcerated youth, (2) compare adolescent users of both NO and VS inhalants (NO+VS) to users of NO-only, VS-only, and nonusers of NO and VS (NO/VNS nonusers) with regard to demographic, psychological, and behavioral characteristics, and (3) conduct logistic regression analyses identifying correlates of NO use. Residents (N = 723) of Missouri Division of Youth Services were assessed with standardized psychosocial measures. Participants averaged 15.5 (SD = 1.2) years of age, were ethnically diverse and predominantly male. Lifetime prevalence of NO use was 15.8%. NO+VS users evidenced greater impairments compared to NO+VS nonusers. VS-only users evidenced impairments that were similar in kind but at lower prevalences compared to those displayed by NO+VS users, whereas NO-only youth had profiles that were similar to those of NO/VS nonusers. Psychiatric disorders, polydrug use, and temperamental fearlessness were correlates of NO use. NO+VS users were at high risk for behavioral and emotional problems. Screening and interventions for NO and VS inhalant use should be implemented in juvenile justice facilities.Peer Reviewedhttp://deepblue.lib.umich.edu/bitstream/2027.42/78160/1/nihms217666.pd