Rare Diseases, Drug Development and AIDS: The Impact of the Orphan Drug Act

In this article, we examine the Orphan Drug Act with an eye toward its contribution to the public interest, using AIDS drugs to illustrate many of the central points. The major policy question is, How, if at all, can the act be used to meet the legislative goal of stimulating drug development for small patient populations without resulting in prices that make drugs inaccessible

Rare Diseases, Drug Development and AIDS: The Impact of the Orphan Drug Act

In this article, we examine the Orphan Drug Act with an eye toward its contribution to the public interest, using AIDS drugs to illustrate many of the central points. The major policy question is, How, if at all, can the act be used to meet the legislative goal of stimulating drug development for small patient populations without resulting in prices that make drugs inaccessible

What Predicts Intent to Breastfeed Exclusively? Breastfeeding Knowledge, Attitudes, and Beliefs in a Diverse Urban Population

Maternal knowledge and comfort with breastfeeding affect prenatal feeding intentions, and these intentions are strong predictors of feeding outcomes. However, predictors of exclusive breastfeeding intention have not been well characterized

Effect of Primary Care Intervention on Breastfeeding Duration and Intensity

Objectives. We determined the effectiveness of primary care–based, and pre- and postnatal interventions to increase breastfeeding. Methods. We conducted 2 trials at obstetrics and gynecology practices in the Bronx, New York, from 2008 to 2011. The Provider Approaches to Improved Rates of Infant Nutrition & Growth Study (PAIRINGS) had 2 arms: usual care versus pre- and postnatal visits with a lactation consultant (LC) and electronically prompted guidance from prenatal care providers (EP). The Best Infant Nutrition for Good Outcomes (BINGO) study had 4 arms: usual care, LC alone, EP alone, or LC+EP. Results. In BINGO at 3 months, high intensity was greater for the LC+EP (odds ratio [OR] = 2.72; 95% confidence interval [CI] = 1.08, 6.84) and LC (OR = 3.22; 95% CI = 1.14, 9.09) groups versus usual care, but not for the EP group alone. In PAIRINGS at 3 months, intervention rates exceeded usual care (OR = 2.86; 95% CI = 1.21, 6.76); the number needed to treat to prevent 1 dyad from nonexclusive breastfeeding at 3 months was 10.3 (95% CI = 5.6, 50.7). Conclusions. LCs integrated into routine care alone and combined with EP guidance from prenatal care providers increased breastfeeding intensity at 3 months postpartum

A Cluster Randomized Trial of Tailored Breastfeeding Support for Women with Gestational Diabetes

Background: Women with gestational diabetes mellitus (GDM) and their infants are at increased risk of developing metabolic disease; however, longer breastfeeding is associated with a reduction in these risks. We tested an intervention to increase breastfeeding duration among women with GDM

Parents’ Beliefs Regarding Shared Reading with Infants and Toddlers

Parent beliefs about reading to young children- and factors related to such beliefs- affect a child’s reading skill. But, little is known about parent beliefs about reading to infants and toddlers. To fill this gap, three University Centers of Excellence in Developmental Disabilities (UCEDDs) studied 43 English and Spanish speaking parents of children aged 9-18 months. The three UCEDDs were working on a project to create a children’s book that had tips for parents about how their one year-old learns and grows. The UCEDD study survey asked about parent beliefs about reading to young children (4 questions) and factors related to those beliefs (2 questions). Parents were also asked to give feedback about the book. Nearly all parents agreed that children should be read to as infants and that this helps children develop reading skills. Most (62%) parents said it was ‘very common’ for friends and family to read with children of this age (62%). Parents said that reading the board-book together was: useful for “promot[ing] language,” “help[ing] my baby’s development,” and “help[ing] my child speak.” More research like this can identify ways to help parents of young children develop reading skills

Association of parent-child interactions with parental psychological distress and resilience during the COVID-19 pandemic

IntroductionThe effects of psychological distress/resilience on parent-child engagement (e.g., family dinners, reading) during the COVID-19 pandemic have not been well studied. Among very young children from underrepresented backgrounds enrolled in the ongoing longitudinal Bronx Mother Baby Health Study of healthy term infants, we (1) examined associations between exposures to COVID-19-related events, demographic factors and parental psychological distress and resilience; and (2) correlated these factors with parent-child engagement activities.MethodsBetween June 2020-August 2021, parents of 105 Bronx Mother Baby Health Study participants aged birth-25 months completed questionnaires related to exposures to COVID-19-related events, frequency of positive parent-child engagement activities, food and housing insecurity, and parental psychological distress and resilience. Families were also asked open ended questions about the pandemic's impact.Results29.8% and 47.6% of parents reported food and housing insecurity, respectively. Greater exposures to COVID-19-related events were associated with increased parental psychological distress. Positive parent-child interactions were associated with demographic factors and higher levels of maternal education, but not with exposures to COVID-19-related events.DiscussionThis study adds to a growing body of literature on the negative impacts of COVID-19 exposures and psychosocial stressors on families during the pandemic, supporting the need for enhanced mental health resources and social supports for families

Rationale, design, and methodology for the optimizing outcomes in women with gestational diabetes mellitus and their infants study

Background Women who are diagnosed with gestational diabetes mellitus (GDM) are at increased risk for developing prediabetes and type 2 diabetes mellitus (T2DM). To date, there have been few interdisciplinary interventions that target predominantly ethnic minority low-income women diagnosed with GDM. This paper describes the rationale, design and methodology of a 2-year, randomized, controlled study being conducted in North Carolina. Methods/Design Using a two-group, repeated measures, experimental design, we will test a 14- week intensive intervention on the benefits of breastfeeding, understanding gestational diabetes and risk of progression to prediabetes and T2DM, nutrition and exercise education, coping skills training, physical activity (Phase I), educational and motivational text messaging and 3 months of continued monthly contact (Phase II). A total of 100 African American, non-Hispanic white, and bilingual Hispanic women between 22–36 weeks of pregnancy who are diagnosed with GDM and their infants will be randomized to either the experimental group or the wait-listed control group. The first aim of the study is to determine the feasibility of the intervention. The second aim of study is to test the effects of the intervention on maternal outcomes from baseline (22–36 weeks pregnant) to 10 months postpartum. Primary maternal outcomes will include fasting blood glucose and weight (BMI) from baseline to 10 months postpartum. Secondary maternal outcomes will include clinical, adiposity, health behaviors and self-efficacy outcomes from baseline to 10 months postpartum. The third aim of the study is to quantify the effects of the intervention on infant feeding and growth. Infant outcomes will include weight status and breastfeeding from birth through 10 months of age. Data analysis will include general linear mixed-effects models. Safety endpoints include adverse event reporting. Discussion Findings from this trial may lead to an effective intervention to assist women diagnosed with GDM to improve maternal glucose homeostasis and weight as well as stabilize infant growth trajectory, reducing the burden of metabolic disease across two generations. Trial registration NCT0180943

The influence of snoring, mouth breathing and apnoea on facial morphology in late childhood: a three-dimensional study

Objective: To explore the relationship between the prevalence of sleep disordered breathing (SDB) and face shape morphology in a large cohort of 15-year-old children. Design: Observational longitudinal cohort study Setting: Avon Longitudinal Study of Parents and Children (ALSPAC), South West of England. Participants: Three-dimensional surface laser scans were taken for 4784 white British children from the ALSPAC during a follow-up clinic. A total of 1724 children with sleep disordered breathing (SDB) and 1862 healthy children were identified via parents’ report of sleep disordered symptoms for their children. We excluded from the original cohort all children identified as having congenital abnormalities, diagnoses associated with poor growth and children with adenoidectomy and/or tonsillectomy. Main outcome measures: Parents in the ALSPAC reported sleep disordered symptoms (snoring, mouth breathing and apnoea) for their children at 6, 18, 30, 42, 57, 69 and 81 months. Average facial shells were created for children with and without SDB in order to explore surface differences. Results: Differences in facial measurements were found between the children with and without SDB throughout early childhood. The mean differences included an increase in face height in SDB children of 0.3 mm (95% CI −0.52 to −0.05); a decrease in mandibular prominence of 0.9° (95% CI −1.30 to −0.42) in SDB children; and a decrease in nose prominence and width of 0.12 mm (95% CI 0.00 to 0.24) and 0.72 mm (95% CI −0.10 to −0.25), respectively, in SDB children. The odds of children exhibiting symptoms of SDB increased significantly with respect to increased face height and mandible angle, but reduced with increased nose width and prominence. Conclusions: The combination of a long face, reduced nose prominence and width, and a retrognathic mandible may be diagnostic facial features of SBD that may warrant a referral to specialists for the evaluation of other clinical symptoms of SDB