The approval was supported by data from ARRIVAL (N=25), an ongoing Phase 3 open-label safety study in children with CF aged 12 to <24 months with 1 of 10 CFTR gene mutations (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D or R117H).
The indication was approved under an accelerated approval pathway based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate.
The authors report that current data do not support greater dietary diversity as an effective strategy to promote healthy eating patterns and healthy body weight.
Researchers identified 23 randomized-controlled trials that included patients with PCOS and overweight/obesity (N=941) and compared the effectiveness of various pharmacological interventions (i.e., metformin, inositol, liraglutide, orlistat) on weight reduction.
A comparative list of medications by pharmacological class for the treatment of obesity.
Five Phase 1 and three Phase 2 clinical trials of DCCR have been completed, with one of the Phase 2 trials involving PWS patients.
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.