Genetic therapies and respiratory outcomes in patients with neuromuscular disease.

PURPOSE OF REVIEW: Genetic therapies made a significant impact to the clinical course of patients with spinal muscular atrophy and Duchenne muscular dystrophy. Clinicians and therapists who care for these patients want to know the changes in respiratory sequelae and implications for clinical care for treated patients. RECENT FINDINGS: Different genetic therapy approaches have been developed to replace the deficient protein product in spinal muscular atrophy and Duchenne muscular dystrophy. The natural history of these conditions needed to be understood in order to design clinical trials. Respiratory parameters were not the primary outcome measures for the clinical trials. The impact of these therapies is described in subsequent clinical trial reports or real-world data. SUMMARY: Genetic therapies are able to stabilize or improve the respiratory sequelae in patients with spinal muscular atrophy and Duchenne muscular dystrophy. Standardized reporting of these outcomes is needed to help inform the future revisions of clinical standards of care and practice guidelines.

Socioeconomic Status and Determinants of Pediatric Antibiotic Use.

Introduction. Evidence suggests that early-life antibiotic use can alter gut microbiome, predisposing children to obesity. The obesity epidemic has a disproportionate effect on individuals from lower socioeconomic status (SES). Thus, this study aims to explore the link between SES and antibiotic use. Design. We performed a retrospective cohort study of all babies born at and receiving 2 or more outpatient visits at a large, suburban health system in Illinois (United States) between 2007 and 2017. We collected data on zip code as a proxy for SES and antibiotic use in the first year of life. We also obtained comorbid diagnosis codes, race/ethnicity, gender, and number of inpatient, outpatient, and emergency department visits. Results. A total of 7224 patients met our study criteria. Children from low-poverty areas received a lifetime average of 4.28 prescriptions, while those from high-poverty neighborhoods received an average of 3.31 prescriptions. This was statistically significant in our unadjusted analysis but not after adjusting for covariates. Children from high-poverty areas were significantly more likely to receive more antibiotics at 48 hours, 1 week, and 1 month of life in our unadjusted analysis, but not after adjusting for covariates. In our unadjusted and adjusted analyses, children from high-poverty areas were significantly more likely to have received antibiotics at 1 week of life. Conclusion. The relationship between SES and antibiotic use warrants further investigation to help elucidate possible causes of the disproportionate impact obesity has in low-income communities.