MAR 24, 2019 07:39 PM PDT

Injectable Medicine Improves Genetic Rickets

WRITTEN BY: Nouran Amin

A recent study found that an injectable medicine called ‘Burosumab’ was shown to improve symptoms of X-linked hypophosphatemia (XLH)--an inherited form of rickets. According to the Food and Drug Administration (FDA), XLH affects roughly 3,000 children and 12,000 adults in the United States. Symptoms of XLH not only includes rickets, but also bowed legs, bone pain and short stature.

The study demonstrated that burosumab was successful in treating the XLH symptoms in comparison to conventional therapy which includes multiple daily doses of phosphate and vitamin D (e.g. calcitriol).

"These improvements with burosumab have the potential to change the lives of children with XLH as they grow," said study principal investigator Erik Imel, M.D., associate professor of medicine and pediatrics at Indiana University School of Medicine in Indianapolis, Ind.

Individuals with XLH have increased levels of the hormone fibroblast growth factor 23, or FGF23, which results in low blood levels of phosphorus (hypophosphatemia). In the study, patients were either assigned to continue on the conventional therapy or switch to receive the burosumab injections given every two weeks. Improvements were scored by X-rays that measured the degree of rickets along with improvements in in leg deformities, height and distance walked, and an increase in serum phosphorus and active vitamin D levels.

“By 40 weeks of treatment, that improvement was more than two times greater for the burosumab group than the conventional therapy group,” Imel noted. "We now know the magnitude of benefit from the new medication, burosumab, versus the prior approach with conventional therapy. This information is critical for doctors making treatment decisions for their patients with XLH."

Source: Medical Xpress

About the Author
  • Nouran enjoys writing on various topics including science & medicine, global health, and conservation biology. She hopes through her writing she can make science more engaging and communicable to the general public.
You May Also Like
NOV 17, 2019
Drug Discovery & Development
NOV 17, 2019
Drug Reduces Heart Attacks in Diabetic Patients
Recent results from a clinical trial that evaluated the addition of a drug called ‘ticagrelor’ to aspirin were shown to improve clinical outcom...
NOV 17, 2019
Drug Discovery & Development
NOV 17, 2019
Does TruBrain Really Work?
TruBrain is a mix of different substances claimed to help people increase verbal fluency, avoid distractions, and boost their mental output. Created by a t...
NOV 17, 2019
Drug Discovery & Development
NOV 17, 2019
Researchers Can Now Reverse Skin Cancer
Ten years ago, just 5% of people with advanced melanoma (skin cancer) lived more than five years after being diagnosed. Now however, researchers from the I...
NOV 17, 2019
Drug Discovery & Development
NOV 17, 2019
Drug Increases Survival Rates for Heart Failure Patients
Scientists have demonstrated in preclinical studies that a drug called ‘Aliskiren’ works by inhibiting the activity of an enzyme involved in bl...
NOV 17, 2019
Microbiology
NOV 17, 2019
The Antimicrobial Power of Mucus is Revealed
We produce several liters of mucus every day to cover more than 200 square meters in the human body....
NOV 17, 2019
Immunology
NOV 17, 2019
Treating Celiac Disease May Be Possible
Delivering gluten to the body in a friendly, harmless package may be the way for people with celiac disease to finally have a sandwich again. A new clinica...
Loading Comments...