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90-day interim biopsy data in the first 3 patients revealed an average microdystrophin expression of 110% and improvements in muscle health and resilience biomarkers.

Nurse practitioner Donna Hallas, PhD, PPCNP-BC, CPNP, PMHS, FAANP, FAAN offers her thoughts on the recent FDA approval of GSK's meningococcal ABCWY vaccine.

Get caught up with Contemporary Pediatrics! This list helps you navigate our top stories from the week, all in one place.

The combination vaccine targets 5 groups of the bacteria Neisseria meningitidis (A,B,C,W, AND Y) that cause the most invasive meningococcal disease cases (IMD) globally.

The federal agency has approved Merilog (insulin-aspart-szjj) as biosimilar to Novolog (insulin aspart) for the improvement of glycemic control in adults and pediatric patients with diabetes mellitus.

Genetic research is revealing that up to 35% of cerebral palsy cases have an identifiable genetic cause, leading to earlier diagnoses and potential precision medicine treatments.

Higher physical activity and lower screen time in childhood were linked to reduced stress and depression in adolescence, according to a Finnish cohort study.

Peter S. Jensen, MD, comments on the challenges primary health care providers may face when it comes to mental health treatment in pediatrics, and provides screening tools to consider to combat these challenges.

Ensoma's lead program, EN-374, has been granted a pair of FDA designations for the treatment of X-linked chronic granulomatous disease.

His mother reported that the nail had developed a green discoloration and had begun to detach approximately 1 week prior to the visit.

Efficacy for the kinase inhibitor was based on the ReNue (NCT03962543 ) trial that featured 58 adults and 56 pediatric patients.

A review of current guidelines in treatment and thromboprophylaxis.

M pneumoniae infection is not a reportable disease, and testing is often not performed in the outpatient setting, limiting estimates of clinical prevalence and delaying recognition of community epidemics.

"Less pain and no need for sedation are major benefits," said Jon Matthew Farber, MD, in this edition of Journal Club.

In this discussion, leading experts weigh in on the importance of scientific integrity, proactive advocacy, and the crucial role of vaccinations in pediatric health.

Get caught up with Contemporary Pediatrics! This list helps you navigate our top stories from the week, all in one place.

The investigational biologic targets the interleukin-2 receptor complex in the body in order to stimulate proliferation of inhibitory immune cells.

Shriners Children’s Boston warns patients and their parents about frostbite risks, offering tips on prevention, signs, and treatment to keep kids safe in extreme cold.

Cyclo Therapeutics has enrolled 10 patients in a single-arm sub-study treating newborns to 3 years of age, evaluating the Trappsol Cyclo in the youngest subsets.

A look into nutrition's role in acne care, highlighted by Colleen Sloan, PA-C, RDN.

Allergic sensitization was more prominent in severe phenotypes but did not explain seasonal differences.

The FDA is warning diabetes patients that information from CGMs, insulin pumps, and automated dosing systems could fail to be delivered if smartphone settings are not properly configured.

The designations follow the FDA acceptance of IND application for ABO-101 to treat PH1, with a phase 1/2 study planned in the first half of 2025.

A recap of the FDA submissions and regulatory decisions in pediatrics from January 2025.

"I’m all for reducing opioids, especially in neonates, for whom we are unsure of long-term adverse effects," stated Jon Matthew Farber, MD.

The primary and all key secondary endpoints were met compared to cream vehicle in the phase 3 DELTA TEEN trial.

Application submission is based on positive phase 3 data that demonstrated improvement in patients with type 2 and 3 SMA receiving current standard of care.