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US okays drug to improve growth in children with dwarfism

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In this file photo taken on July 20, 2020, a sign for the Food And Drug Administration is seen outside of the headquarters in White Oak, Maryland. (Photo by Sarah Silbiger / GETTY IMAGES NORTH AMERICA / AFP)

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.

 

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Oyo: Tragedy As Bride-to-Be Among Four Dead in Suspected Lassa Fever Outbreak

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The Oyo State Ministry of Health has closed two hospitals in Saki West Local Government Area following a suspected outbreak of Lassa fever, which has claimed four lives.

The fatalities, confirmed by the Oyo State Rapid Response Team (RRT), include three males and a 32-year-old female who was preparing for her wedding. The closure of the hospitals aims to contain the spread of the disease and ensure thorough decontamination.

In a press statement issued by the Commissioner for Information and Orientation, Dotun Oyelade, the State Commissioner for Health, Dr. Oluwaserimi Ajetunmobi, revealed that the RRT was dispatched to Saki following distress calls from residents reporting mysterious deaths.

Findings and Immediate Actions

The RRT, comprising health officials, representatives from WHO and the Red Cross, and local health authorities, conducted a comprehensive investigation into the outbreak.

Preliminary findings suggest that an apprentice at one of the affected hospitals, who had recently returned from Iwajowa Local Government Area, might have introduced the infection.

The team disclosed that the deceased exhibited symptoms consistent with Lassa fever, such as craniofacial bleeding. The hospital owner, who was among the deceased, initially self-medicated with antimalarials and antibiotics before seeking hospital care when his condition worsened.

Containment Measures

Both the hospital where the fatalities occurred and the facility that admitted the hospital owner were shut down for decontamination.

Contact tracing efforts are ongoing, focusing on high-risk individuals who had contact with the deceased during their illness or burial.

Affected health workers were trained on Infection Prevention and Control (IPC) protocols, and public awareness campaigns were conducted to educate residents about Lassa fever.

Samples have been collected from a symptomatic individual, pending laboratory confirmation. Line-listing of contacts is underway to identify potential cases and prevent further spread.

Collaborative Efforts

The RRT also paid an advocacy visit to the Okere of Saki’s palace to brief traditional authorities on the situation. Local government officials in Saki West were engaged to coordinate containment strategies.

Reports indicate that while the outbreak initially affected Saki West, it has extended to Iwajowa and Kajola Local Government Areas.

Disease Surveillance and Notification Officers (DSNOs) in the affected LGAs have been instructed to commence preliminary containment measures as the State RRT ramps up its response.

Call for Vigilance

The Ministry of Health has urged residents to remain vigilant, report any symptoms suggestive of Lassa fever, and adhere to preventive measures.

These include proper hygiene practices, avoiding contact with rodents, and seeking medical attention promptly if symptoms arise.

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Oyo: Oseni to Launch Mobile Clinic Initiative to Revitalize Rural Healthcare

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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.

 

 

 

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NAFDAC Shuts Maiduguri Market Over Sun-Dried Drugs After Flood

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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.

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