
Lomitapide: A Bright Hope for Kids with Homozygous Familial Hypercholesterolemia (HoFH)
Living with homozygous familial hypercholesterolemia (HoFH) can feel overwhelming. This rare genetic condition causes dangerously high LDL cholesterol levels from a young age. For children, it means facing the risk of heart disease or stroke far too early in life. However, lomitapide, a medication originally approved for adults in 2012, is showing promising results for kids with HoFH.
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Breaking New Ground: Liraglutide Offers Hope for Childhood Obesity in Kids Under 12
The SCALE Kids study showed that liraglutide helped children aged 6 to under 12 with obesity reduce their BMI when combined with lifestyle changes like better eating and more exercise. Children taking liraglutide had better results than those who took a placebo, with many achieving meaningful weight loss.
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Categorized as Child Health, Dr. Tashko’s Key Clinical Trials, Drug Information, GLP1 agonist, Obesity Care
Closed-Loop Insulin Pump for Children Ages 2-6
March 2023 NEJM The PEDAP trial. The Pediatric Artificial Pancreas (PEDAP) clinical trial was conducted to compare the efficacy and safety of closed-loop insulin delivery with standard care in children with type 1 diabetes ages 2-6. Closed-loop insulin delivery is a system that automatically delivers insulin based on real-time glucose measurements. Standard care consisted of either an insulin pump or multiple daily injections of insulin plus a continuous glucose monitor. The closed-loop insulin delivery system used…
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A great prospect for type 1 diabetes
Type 1 diabetes is a devastating life-long condition. It is autoimmune and frequently occurs in predisposed young adults. Chronic therapy is insulin use via multiple daily injections or insulin-pump; in addition to intensive lifestyle modifications. The best management of type 1 diabetes is proactive prevention or elimination rather than reactive insulin usage. The current study addresses this very point. A group of 76 participants at high-risk for DM1 was randomized to receive teplizumab or placebo.…
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Victoza use in children with diabetes type 2
Results of ELLIPSE trial are of major significance as they show that Victoza addition to Metformin helps in further reduction of A1c and fasting plasma glucose. The efficacy and safety appear to be similar as in adults. The most common side effects are gastrointestinal in nature. Based on ELLIPSE trial outcomes, I anticipate broadening of FDA indications for Victoza, to include children and adolescents GT
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Pituitary enlargement due to hypothyroidism
Another nice report of total pituitary enlargement due to the long-standing untreated primary hypothyroidism. Low levels of FT3 and FT4 lead to excessive TRH and TSH production, in turn causing pituitary expansion. This case is unique as it is found in a child suffering from growth retardation. Proper treatment with thyroid hormone supplementation reversed the illness. GT
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Novel Approach: Verapamil for Type 1 Diabetes
Old drug, new approach. Verapamil has classically been used to treat hypertension and cardiac arrhythmias. This randomized phase 2 clinical trial shows a novel use of the drug by improving pancreatic beta cell function in recently diagnosed type 1 diabetes. Verapamil helped patients use lower doses of insulin as well as experiencing less hypoglycemia. These are exciting and promising results. GT
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Stop puberty obesity!
This is a nice epidemiological study of childhood overweight and obesity as it pertains to development of type 2 diabetes in adulthood. Authors have observed that heavy children who are no longer overweight after age 13 are not at increased risk of diabetes as adults. On the contrary, the longer the duration of high BMI during puberty, the higher the probability of developing early diabetes mellitus. GT
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New FDA approval: the first drug for x-linked hypophosphatemia
Crysvita or burosumab is the first drug to be approved by FDA for treatment of x-linked hypophosphatemia in adults and children older than one year of age. It is inherited, rare and unresponsive to vitamin D supplementation. It is a form of ricket and osteomalacia leading to low blood phosphorus levels. Clinical manifestations are disabled bone growth and development in children and impaired bone mineralization in adults. GT
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Categorized as Blog, Child Health, FDA Approvals, Insights from Dr. Tashko, Osteoporosis Care & Prevention
Trends of kidney disease in children with type 1 diabetes
This observational study reveals important long-term trends of type 1 diabetes in regard to kidney function & end stage renal disease (ESRD). Subjects were followed for up to 42 years: Rates of ESRD are lower when children are diagnosed with DM1 before age 10 than during puberty. Kidney disease is less prevalent in females than males. Diabetic nephropathy peaks at 25 years after diagnosis and remains stable subsequently. These findings could be shared with parents…
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Albumin excretion during puberty in the setting of type 1 diabetes.
The use of an ACE inhibitor and a statin did not change the albumin-to-creatinine ratio over time among adolescents with type 1 diabetes. Neither drug had significant effects on carotid intima–media thickness, other cardiovascular markers, the glomerular filtration rate, or progression of retinopathy. NEJM
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