Publications by authors named "R Horikawa"

Context: Several long-acting growth hormone (LAGH) therapies have recently become available, but guidance on their usage in children with growth hormone (GH) deficiency is limited.

Methods: International experts in pediatric endocrinology were invited to join a consensus group based on their expertise in treating children with daily GH and LAGH. The group comprised 11 experts from 10 countries across the world.

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Article Synopsis
  • Liver transplantation (LDLT) can effectively treat children with propionic acidemia (PA), showing a 100% survival rate in a study of 12 patients.
  • The study compared outcomes based on the timing of LDLT, dividing patients into groups based on whether the surgery occurred within a year of their first metabolic decompensation or later.
  • Early LDLT may prevent complications like cardiomyopathy and neurological issues, despite later patients showing some pre-existing conditions and post-surgery developmental challenges.
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  • Somapacitan, a weekly growth hormone treatment, was tested in a study involving short children born small for gestational age (SGA) to evaluate its efficacy and safety compared to daily growth hormone.
  • The study involved 62 participants across 38 sites globally, with findings showing that higher doses of somapacitan led to significant growth in height compared to daily GH, with similar results regarding insulin-like growth factor levels.
  • After 52 weeks, somapacitan at 0.24 mg/kg/week was found to be as effective, safe, and well-tolerated as the higher daily GH dose of 0.067 mg/kg/day for promoting growth in these children.
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