Child & Adolescent Development: Overview
Resources
Basic InformationMore InformationLatest News
Unexplained Drop in Resting Heart Rate in Youth 'Not a Good Thing'Strike Out Kids' Overuse Injuries This Baseball SeasonMost Young Americans Eager to Get COVID Vaccine: PollMany Kids Who Develop Severe COVID-Linked Syndrome Have Neurologic SymptomsMost Parents OK About School Rules for Kids' Return to Sports: PollSome Kids Snore, and It Could Affect BehaviorKids With Autism Can Really Benefit From ExerciseFDA Approves First New Children's ADHD Drug in 10 YearsWhy Are ER Wait Times Getting Longer for Kids in Mental Health Crisis?About 40,000 U.S. Children Have Lost a Parent to COVID-19Adding in Stem Cell Therapy Helps Beat a Common Childhood LeukemiaWhat Will Summer Camp Look Like This Year?When Will America's Kids Get Their COVID Vaccines?1 in 4 Parents Won't Vaccinate Their Kids Against COVID-19: PollEven in a Pandemic, Child Vision Tests Are CrucialPfizer Says Its COVID Vaccine Is Very Effective in Kids as Young as 12Secondhand Smoke Is Sending Kids to the ERDrug Shows Promise Against Rare Condition That Stunts Kids' GrowthWhen Coal-Fired Power Plants Close, Kids With Asthma Breathe EasierAnother Study Finds COVID Doesn't Spread in Schools With Proper SafeguardsNearly Half of U.S. Schools Now Offer In-Person LearningLockdowns Gave Boost to Type 1 Diabetes Control in KidsWildfire Smoke Can Send Kids With Asthma to the ERPandemic Has Many Kids Struggling With Weight IssuesLab-Made Heart Valves Can Grow Along With Youngest Heart PatientsSome Kids With Type 1 Diabetes Face High Risk of Severe COVID-19Virtual Learning Has Taken a Toll on Kids' & Parents' Mental HealthCDC Says 3 Feet of Social Distancing Now OK in Most ClassroomsWhich Kids' Sports Have Higher Odds for Head Injury?Social Distancing Probably Stopped 2020 Outbreak of Paralyzing Disorder in KidsAHA News: What Parents Should Know About Rare But Scary COVID-19-Related IllnessSchool Dental Care Program Could Cut Cavities in Half: StudySocial Media, Binge Eating Often Go Together for KidsStressed and Distracted, Kids and Their Teachers Say Virtual Learning Isn't WorkingSports Position Doesn't Affect Risk of Concussion-Linked CTE IllnessPandemic Putting Added Strain on Parents of Kids With CancerDogs and Kids Are 'In Sync,' Study ShowsTeachers Main Drivers of School COVID Outbreaks, So Vaccinations Needed: StudyTips to Keep Young Athletes Injury-FreeMental Illness in Childhood Could Mean Worse Physical Health Decades LaterKids' Robust Immune Systems May Shield Them From COVID-19: StudyFertility Treatments Might Affect Kids' Growth, But Not for LongMom's Heart Health While Pregnant Could Influence Her Child's Health for YearsPandemic Has Affected Kids' Dental Health: PollNew Rabies Prevention Treatment Also Works in Kids: StudyWhen Will Kids Get the COVID Vaccines?U.S. Schools Can Reopen, With Safeguards in Place: CDCFetal Surgery Is Changing Lives for Kids With Spina BifidaKids Who Got Flu Shot Had Milder COVID Symptoms: StudyVery Little Spread of Coronavirus at Kids' Day Camps: Study
Questions and AnswersVideosLinksBook Reviews
Related Topics

ADHD: Attention Deficit Hyperactivity Disorder
Childhood Mental Disorders and Illnesses
Parenting
Child Care

Drug Shows Promise Against Rare Condition That Stunts Kids' Growth

HealthDay News
by By Alan Mozes HealthDay Reporter
Updated: Mar 25th 2021

new article illustration

THURSDAY, March 25, 2021 (HealthDay News) -- A new medication may offer hope to children with achondroplasia, a rare bone growth disorder that causes very short stature coupled with disproportionate limb and trunk size.

The experimental drug is called vosoritide. By tamping down overactive growth plate signaling that impedes bone growth, the drug seeks to offer affected children the possibility of greater height and improved proportions.

"Currently, there are no treatments that are targeted at its source," said study author Dr. Ravi Savarirayan. Left untreated, patients experience medical and functional complications, he said, including spinal column narrowing (spinal stenosis), spinal cord compression and bowed legs.

But a daily dose of the investigational drug -- administered to nearly 120 children for two years -- translated into height gains among patients 5 to 18 years old.

Prior to the study, the children had gained an average of 1.67 inches in height a year. But one year into the trial, yearly height gains averaged 2.23 inches. The gains continued over a second year of treatment, dipping slightly to 2.19 in additional height a year, on average.

"We found daily treatment increased growth to the rate approximating those without the condition," said Savarirayan, a professor and clinical geneticist with the Murdoch Children's Research Institute at Royal Children's Hospital in Parkville, Australia.

The drug also appeared to improve body proportions and was safe and durable, meaning the benefit did not appear to wane over time.

The study team noted that achondroplasia is the most common driver of disproportionate short stature. Children with the condition tend to have short limbs and average trunk length. Their maximum height is typically under 4 feet 6 inches.

Savarirayan said the genetic condition affects roughly 360,000 people around the world, and that the drug under study is only designed to address this particular condition, not short stature in general.

Dr. Alan Rogol is professor emeritus of pediatrics and pharmacology at the University of Virginia and a former vice president of The Endocrine Society. He said, "This is the most common cause out of at least 1,000 different causes of disproportionate short stature." But it's critical to distinguish between the condition and efforts to treat it, on the one hand, and simply being short on the other, he added.

"Healthy short people are proportionate, and everything works," Rogol noted. "People with this condition have very small hands and feet, a relatively large head, and have a very difficult time with their spine, especially at both ends. And they're more likely to have serious complications, including sleep apnea, obesity and difficulty with activities of daily living and developmental delays."

Many of those concerns can, in fact, be addressed, Rogol said. But for growth itself there has been little in the offing for achondroplasia patients.

"There is growth hormone, but it's only approved in Japan, nowhere else, and if it is effective in making patients taller, it is a minor league effect," he said.

"And then there's limb lengthening, which means breaking the legs, breaking the arms, putting in a metal distractor between the two ends, and cranking it up a few tenths of a millimeter every few days," Rogol said.

"With this, you can get limb lengthening and growth," he acknowledged. "It's a legitimate procedure. There are some good doctors who do this. But it usually goes on six to 12 months, and it's a horrific procedure with tremendous side effects. It's not anything that the faint of heart should do without some serious consideration."

As for vosoritide, Rogol cautioned that "it's not whether it works or not, but how well. There's no question that the children had a height growth velocity that was greater than it was before. But the difference after a year or two is very, very small. It's only marginally significant. So it's a value judgment that the parents are going to have to make."

And there are philosophical considerations as well, he added, given that there's an argument to be made that "little people are fine as they are, are supposed to be little, and don't need anything to be done about it."

According to Savarirayan, vosoritide is under review by both the U.S. Food and Drug Administration and the European Medicines Agency. The research was funded by BioMarin, the drug's maker. The findings were presented Saturday at a virtual meeting of The Endocrine Society.

Data and conclusions presented at meetings should be considered preliminary until published in a peer-reviewed medical journal.

More information

There's more on achondroplasia at the U.S. National Center for Advancing Translational Sciences.

SOURCES: Ravi Savarirayan, MD, professor and clinical geneticist, Murdoch Children's Research Institute at Royal Children's Hospital in Parkville, Australia; Alan D. Rogol, MD, PhD, professor emeritus, pediatrics and pharmacology, University of Virginia, and former vice president, The Endocrine Society; The Endocrine Society, virtual annual meeting, March 22 to 23, 2021




Facebook

Amazon Smile

 

Children and Adult services are available now with no wait time.  

Please contact HBH at 860-548-0101, option 2.

 


powered by centersite dot net