Approximately 12,000 patients took part in the CAMELLIA-TIMI 61 trial in which participants were randomized to either lorcaserin HCl 10mg twice daily or placebo.
"These children don't have to die," KidsAndCars.org founder and president Janette Fennell said in a statement. "Parents and caregivers have the power to make sure that this doesn't happen to them."
For the same 25 cases, a Heat Index screening threshold of 85 degrees Fahrenheit could identify potentially hazardous levels of workplace environmental heat when WBGT was unavailable. Six fatalities occurred when the Heat Index was <91 degrees Fahrenheit.
CUTX-101, a subcutaneous injectable formulation of copper histidinate, is designed to supplement blood and brain copper levels in patients with Menkes disease.
Medicines to treat cancer (50%); metabolic, endocrine, and genetic diseases (17.4%); and infectious diseases (5.2%) were most common in these programs. Ninety-two FDA-approved drugs and biologics with associated expanded access programs initiated prior to FDA approval between September 1996 and June 2017.
Age-adjusted prevalence of obesity was increased for men living in medium or small metropolitan statistical areas (MSAs) versus those living in large MSAs (42.4 vs 31.8%); the difference for men living in large MSAs vs non-MSAs was not statistically significant.
This case details complications related to tattooing in a 31-year-old woman with a history of cystic fibrosis who had undergone bilateral lung transplantation in 2009 and was on long-term immunosuppressive therapy.
Current treatments for MPS 1 include bone marrow transplant and enzyme replacement with a recombinant form of human IDUA administered intravenously.
Compared to women with stable healthy normal-weight, women who maintained metabolically healthy obesity during follow-up were still at elevated cardiovascular risk (hazard ratio, 1.57); this risk was lower than for initially metabolically healthy women who converted to an unhealthy phenotype (hazard ratios, 1.90 and 2.74 for normal-weight and obesity, respectively).
Palynziq is a PEGylated phenylalanine ammonia lyase enzyme that converts phenylalanine to ammonia and trans-cinnamic acid.
During 1,836,452 person-years of follow-up, the incidence of new episodes of ≥5% weight gain was 8.1 and 11.2 per 100 person-years in participants not prescribed and prescribed antidepressants, respectively (adjusted rate ratio, 1.21).
This investigational lentiviral-based gene therapy is currently being studied in the Phase 2/3 Starbeam Study, a 2-year study assessing the safety and efficacy of Lenti-D in boys ≤17 years old with CALD.
Antisense technology refers to the use of synthetic nucleic acid sequences to interrupt specific protein production by targeting the corresponding messenger RNA that encodes that protein.
Higher BMI in lesbian and bisexual women is an important contributor to the greater level of type 2 diabetes among this population, the authors found.
Improved research methods needed to determine effectiveness of anti-obesity public health initiatives
Researchers found that amongst nearly 300 studies reporting the impact of policies, programs, or changes in built environment on controlling and preventing obesity, most natural experiments had high risk of bias and poor handling of dropouts and withdrawals.
The researchers found that at baseline, severely obese patients had higher disability scores versus overweight patients in both Forward and VARA.
In terms of study design, guidance, documentation, and communication about methods used should be improved. Cross-cutting recommendations are provided for improving the rigor of natural experiments in obesity.
Crysvita, a recombinant fully human monoclonal IgG1 antibody, works by blocking fibroblast growth factor 23 (FGF23), a hormone that causes phosphate urinary excretion and suppresses vitamin D production by the kidney.
The researchers found that on average, patients and their parents perceived FBT as "quite helpful" and "extremely helpful," respectively.
Weight stigma was associated with increased odds of having a body mass index (BMI) consistent with underweight or obesity compared with normal weight.
Patients were treated for a median of 2.2 years. The researchers found that the primary cause of death was heart failure (79.4%), when identified. One death (3.7%) occurred among the 27 patients in the first trial group; among the 27 patients in the matched untreated group, there were 9 deaths (33.3%).
The researchers observed no significant difference in the Pmax-to-PVO2max ratio between children and endurance athletes (1.9 ± 0.5 versus 2.1 ± 0.2); the ratio was lower than that of untrained men (3.2 ± 0.3).
"The combination of ethnicity/race with markers of insulin resistance and genetic factors might help identify obese youth at risk for developing fatty liver," the authors write.
Miglustat, a glucosylceramide synthase inhibitor, is indicated as monotherapy for the treatment of adults with mild to moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option (eg, due to allergy, hypersensitivity, or poor venous access).
Crysvita is an antibody that blocks fibroblast growth factor 23 (FGF23), a hormone that causes phosphate urinary excretion and suppresses active vitamin D production by the kidney.
The TAZPOWER study is a randomized, double-blind, placebo-controlled Phase 2/3 crossover study evaluating the safety and efficacy of elamipretide (daily subcutaneous injections) in 12 male patients (aged ≥12 years) with genetically-confirmed Barth syndrome.
Each additional 10cm greater waist circumference at early childhood was associated with 1.99 higher odds of elevated ALT at mid-childhood, in multivariable-adjusted models.