American and British teams led by Drs Kohn (University of California, Los Angeles), Malech (NIH), Williams (Boston Children's) and Trasher (Great Ormond Street Institute of Child Health) published yesterday in Nature Medicine the conclusive results of a gene therapy trial conducted in the United States and Great Britain in 9 patients with X-linked Chronic Septic Granulomatosis (X-CGD), a rare and severe immune dysfunction. Six of them are free of treatment for complications generated by the disease.
Overweight and obese vitamin D-deficient children who took a relatively high dose of vitamin D every day for six months had lower blood pressure and improved insulin sensitivity than their peers who took a lower dose, according to the results of a UPMC Children's Hospital of Pittsburgh clinical trial reported in The American Journal of Clinical Nutrition.
One in every 15,000 children is affected by Prader-Willi syndrome (PWS), a complex, genetic endocrine condition caused by a disorder of chromosome 15. It's non-inheritable, meaning the condition isn't passed down from a family member.
Cephalalgia, the official journal of the International Headache Society, published the article entitled "Cinnarizine and sodium valproate as the preventive agents of pediatric migraine: A randomized double-blind placebo-controlled trial", by Man Amanat, and Mahmoud Reza Ashrafi from the Tehran University of Medical Sciences, Tehran, Iran.
New findings from a clinical trial show that treatment with the immunotherapy drug blinatumomab is superior to standard chemotherapy for children and young adults with high- or intermediate-risk B-cell acute lymphoblastic leukemia that has relapsed.
A drug used for rheumatoid arthritis has moved a step closer to FDA approval for a desperately needed new use.
A new, strawberry-flavored, granule-form medicine may be a solution for treating infants with AIDs.
The largest-ever clinical trial of a medication for pediatric cardiology patients found that an oral drug significantly improved exercise capacity in adolescent patients with severe, congenital single-ventricle heart defects.
It has been recommended that a drug currently used to prevent preterm birth should be removed from the market.
Results from a phase IIa open-label trial and a subsequent phase III open-label extension trial reinforce findings from an earlier short-term trial that adolescents with moderate-to-severe atopic dermatitis, or eczema, can experience significant improvements with dupilumab.
Researchers have been identifying and modifying known phages to fight against infectious bacteria.
A US study on DNA-based and protein HIV vaccines has shown that an antibody and cellular immune response occurs within six weeks.
A study has revealed how cannabidiol works against tetrahydrocannabinol to prevent psychiatric side effects.