Health
US okays drug to improve growth in children with dwarfism
The US Food and Drug Administration on Friday approved for the first time a drug to improve growth in children with the most common type of dwarfism.
Voxzogo, manufactured by American pharmaceutical company BioMarin, is authorized in the US to treat kids older than five with achondroplasia whose growth plates — zones of cartilage at the end of long bones — are still open, meaning they still have the potential to grow.
“With this action, children with short stature due to achondroplasia have a treatment option that targets the underlying cause of their short stature,” said FDA endocrinologist Theresa Kehoe in a press release.
Achondroplasia is a genetic bone growth disorder that blocks the changing of cartilage to bone. Adults with the condition grow to be about four feet tall (1.2 meters) on average.
It can cause health issues including breathing problems, exaggerated spine curvature, obesity, and recurrent ear infections, according to the National Institutes of Health. Life span is usually near normal.
“As a parent of a child with achondroplasia, I see the availability of treatments that impact bone growth as an important step forward,” Amer Haider, the co-founder of nonprofit group Growing Stronger, said in the BioMarin statement.
Growing Stronger is dedicated to improving the quality of medical care for little people through supporting research.
But growing taller is not necessarily the goal of all with achondroplasia.
When BioMarin shared preliminary test results in 2015, dwarfism advocacy organization Little People of America (LPA) emphasized that it aims to celebrate dwarfism as a “valuable contribution to the diversity of the human condition.”
“While we encourage individuals and families to make the decision that is best for them, we stress that emerging treatments are not necessary for people with dwarfism to live engaging, healthy, productive lives,” the LPA statement said.
Year-long study
The safety and efficacy of Voxzogo, or vosoritide, was evaluated in a year-long, double-blind study for participants five years and older with achondroplasia who have open epiphyses, or growth plates, the FDA said.
Kids who received a Voxzogo injection grew on average about 0.6 inches (1.57 centimeters) taller than those who received a placebo over the period of study.
People with achondroplasia have a genetic mutation that causes a growth regulation gene to be overactive, preventing normal bone growth. Voxzogo works by counteracting the mutation.
The most common side effects were injection site reactions, vomiting, and potentially serious decreased blood pressure.
The European Commission authorized the drug to treat still-growing children older than two years in late August this year.
The medicine is expected to be available in the United States by mid-to-late- December, according to BioMarin.
A member representing Ibarapa East/Ido federal constituency of Oyo State at the House of Representatives, Engr. Aderemi Oseni, has announced a plan to launch a “Mobile Clinic” programme aimed at providing essential healthcare services directly to rural communities across the constituency.
Scheduled to begin in the first quarter of 2025, this initiative underscores Oseni’s commitment to expanding healthcare access, particularly for underserved populations.
Speaking on Saturday at the 10th-anniversary celebration of God’s Knot Hospital in Ologuneru, Ibadan, the lawmaker, who also chairs the House Committee on the Federal Roads Maintenance Agency (FERMA), highlighted the programme’s primary objective: bringing healthcare closer to the people.
“Every community deserves attention; healthcare should never be bound by geographical barriers,” Oseni stated.
He explained that the mobile clinics would deliver essential health check-ups, screenings, and preventive care in remote areas, allowing residents to access quality healthcare without traveling long distances.
As part of his broader healthcare agenda, Oseni informed, through a statement by his media aide, Idowu Ayodele, that he has already facilitated the construction of two fully equipped maternity centers within his constituency, both slated for commissioning before December 2024. He also disclosed plans to establish two modern general hospitals in 2025, further strengthening his commitment to building a healthier community.
The lawmaker expressed interest in collaborating with God’s Knot Hospital on the mobile clinic project, calling for partnerships to support sustainable healthcare initiatives that enhance the quality of life for vulnerable populations in the constituency.
“I resonate deeply with the mission of God’s Knot Hospital,” Oseni shared, adding that such partnerships would significantly improve healthcare in Oyo State”.
The anniversary event attracted distinguished guests, including former Ondo State Governor Dr. Olusegun Mimiko, whom Oseni commended for his contributions to healthcare, particularly through the Mother and Child Hospitals that transformed maternal and child healthcare in Ondo State.
Oseni urged philanthropists, government bodies, and international organizations to support healthcare projects like the upcoming mobile clinics, emphasising that the improvement of healthcare services is a shared responsibility.
The National Agency for Food and Drug Administration and Control (NAFDAC) has ordered the immediate closure of the Gomboru drug market in Maiduguri following severe flooding that submerged the area and contaminated the drugs sold within the market.
In a statement posted on its official X handle, NAFDAC disclosed that the decision was made after reports surfaced that contaminated drugs, which had been washed and sun-dried for resale, were being sold at the market.
“After the recent flooding in Maiduguri that submerged the Gomboru drug market where contaminated drugs were washed and sun-dried for resale, the @DGatNAFDAC ordered the immediate closure of the market to conduct a thorough shop-to-shop removal of these compromised drugs,” the agency wrote.
The flooding, which ravaged Maiduguri, was triggered by the rupture of the Alau dam on the Ngadda River, located 20 kilometers (12 miles) south of the city.
The floodwaters claimed at least 30 lives and displaced 400,000 residents, leaving large parts of the city and surrounding areas in devastation.
NAFDAC’s intervention is aimed at safeguarding public health by preventing the sale and consumption of contaminated drugs.
The agency has mobilized teams to inspect the market and ensure the removal of all compromised pharmaceutical products.
Engr. Aderemi Oseni, Chairman of the House Committee on Federal Road Maintenance Agency (FERMA) and the lawmaker representing Ibarapa East/Ido Federal Constituency of Oyo State has expressed deep concern following the recent outbreak of cholera cases in the state.
Cholera, a highly contagious bacterial infection transmitted through contaminated water and food, poses significant health risks, particularly in densely populated areas with inadequate sanitation.
Oseni urged residents of Oyo State, especially those in his constituency, to prioritise hygiene practices and strictly adhere to recommended safety measures, emphasising the critical importance of proactive measures to halt the disease’s spread.
In a statement issued on Monday by his media aide, Idowu Ayodele, in Ibadan, the state capital, the lawmaker underscored the necessity of maintaining clean water sources and practicing proper sanitation methods.
“Cholera outbreaks can be significantly reduced if we all take responsibility for our hygiene habits and ensure our surroundings are clean,” he stated.
The APC chieftain also stressed the urgency of seeking prompt medical attention at the first signs of symptoms such as diarrhea and vomiting, which are characteristic of cholera.
“Early detection and treatment are crucial in combating cholera and preventing its spread. Access to safe drinking water and adequate sanitation are fundamental rights that must be upheld, especially during health crises like this,” he asserted.
Oseni further called for unity and collective action in combating the cholera outbreak.
He expressed confidence that through concerted efforts and vigilant adherence to preventive measures, communities can contain and ultimately overcome this challenging public health issue.
“By working together, maintaining vigilance, and prioritising hygiene practices, we can effectively mitigate the impact of cholera on our communities,” the lawmaker concluded.
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