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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.

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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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Health

Ondo Resident Doctors Begin 14-Day Warning Strike

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Resident doctors at the University of Medical Sciences Teaching Hospital (UNIMEDTH) in Ondo State have initiated a 14-day warning strike, as confirmed by the National Association of Resident Doctors (NARD).

The strike action, led by the association’s President, John Matthew, highlights various grievances, notably the non-payment of seven months’ salaries to new members.

Matthew added that the strike also addresses the non-payment of palliative to House Officers since February, the failure to disburse the February Hazard Allowance despite prior assurances from the state government, and the ongoing shortage of staff due to resignations.

Matthew lamented the drastic decrease in resident doctors, citing a decrease from 150 doctors to a mere 26 in recent months.

In response to the situation, he urged management to prioritise the full payment of arrears owed to members, immediate payment of the February 2024 palliative to house officers, and prompt disbursement of the February hazard allowance.

The association further called for urgent measures to address the critical shortage of clinical manpower within the institution.

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Health

FG Okays N25bn Disbursement to Enhance Healthcare Delivery

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File photo of the Coordinating Minister of Health and Social Welfare, Ali Pate,

The Federal Government has greenlit the allocation of N25 billion to bolster the efforts of the National Primary Healthcare Development Agency (NPHCDA) and the National Health Insurance Authority (NHIA) in advancing healthcare accessibility and quality across Nigeria.

The Coordinating Minister of Health and Social Welfare, Ali Pate, made this known, stressing the steadfast commitment of the current administration towards revamping the healthcare system to achieve Universal Healthcare Coverage (UHC) for all Nigerians.

In a statement by Deworitshe Patricia, a spokesperson for the ministry, Pate emphasised that the government’s strategy for health sector transformation involves the implementation of a Sector Wide Approach (SWAp) and the Health Sector Renewal Investment Programme (NHSRIP).

Addressing the imperative for reforms in Nigeria’s primary healthcare delivery, Pate highlighted the significance of revising the Basic Healthcare Provision Fund (BHCPF) and its associated programmes.

Pate outlined the objectives of the revised guidelines, which include expanding healthcare services for underserved populations, particularly women and children, reducing maternal mortality rates, alleviating out-of-pocket healthcare expenses, and standardizing healthcare quality across primary healthcare facilities nationwide.

“The Ministry, in collaboration with development partners, is dedicated to ensuring equitable access to healthcare services for all Nigerians, with a special focus on vulnerable groups,” stated Pate. “Through initiatives like NHSRIP, we aspire to minimize the need for long-distance travel for medical care, thereby improving the overall health outcomes and well-being of our citizens.”

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Photos: Saudi Medical Team Make History, Separate Nigerian Conjoined Twins

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In a monumental achievement for medical science, a team of Saudi professionals at the King Abdullah Specialist Children’s Hospital has completed the separation surgery of Nigerian conjoined twins, Hassana and Husaina.

The directive for this historic achievement came from the Custodian of the Two Holy Mosques King Salman bin Abdulaziz Al-Saud and His Royal Highness Prince Mohammed bin Salman bin Abdulaziz Al-Saud, Crown Prince and Prime Minister, demonstrating the Kingdom’s commitment to advancing healthcare.

The complex procedure, initiated on Thursday morning, was the result of meticulous planning and execution by a dedicated team of 38 medical experts. This group included consultants, specialists, technicians, and nursing staff, all working in harmony to address shared areas in the lower abdomen, pelvis, lower spine, and lower spinal nerves of the twins.

The surgery, unfolding in nine carefully planned stages, lasted approximately 14 hours. Dr. Abdullah Al Rabeeah, Advisor at the Royal Court, Supervisor General of the King Salman Humanitarian Aid and Relief Center (KSrelief), and head of the medical team, expressed confidence in the success of the procedure, citing a noteworthy 70% success rate.

“This is a momentous occasion that showcases the Kingdom’s commitment to advancing medical science and providing compassionate healthcare globally,” he said.

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The successful separation of Hassana and Husaina commemorates the 60th operation conducted by the Saudi programme for separating conjoined twins. Over the past 34 years, this programme has offered care to 135 conjoined twins from 25 countries.

Dr. Al Rabeeah expressed heartfelt gratitude and appreciation to the Saudi leadership for their steadfast support of the programme.

He said, “The successful separation of Hassana and Husaina exemplifies the Kingdom’s dedication to humanitarian causes and its role as a leader in global healthcare.”

The Press Attaché at the Saudi embassy in Abuja, Mohammed Alsahabi, remarked on the accomplishment, stating, “This remarkable feat underscores the strong collaboration between Saudi Arabia and Nigeria in healthcare. We take pride in contributing to the improvement of these twins’ lives and their families.”

The separation of Hassana and Husaina represents not just a medical triumph but also serves as a symbol of hope for numerous individuals confronting similar challenges globally.

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