A life-saving medicine for children with Batten disease, a rare and fatal genetic disorder, is set to be pulled from the market because of unresolved pricing negotiations. Otherwise called CLN2, Batten disease causes devastating symptoms, including seizures, loss of mobility, blindness, and worsening dementia, with a life expectancy of around 10 to 12 years. In the UK, approximately 40 children are currently undergoing treatment under a Managed Access Agreement (MAA) between NHS England and BioMarin, the pharmaceutical company that makes the drug. But with the agreement about to end, families worry about losing the sole existing treatment that will slow the disease's progression.

Batten disease is one of the rarest and most destructive neurodegenerative illnesses in children. The inherited disease results in a buildup of waste products in brain cells, producing worsening neurological deterioration, seizures, loss of speech, blindness, and premature death. With no treatment and limited therapies, Batten disease is a devastating diagnosis for families everywhere.

What is Batten Disease?

Batten disease, or neuronal ceroid lipofuscinosis (NCL), is a term used to describe a series of lethal genetic disorders that affect the body's ability to remove waste from cells. Toxic compounds then build up in brain cells and cause them to malfunction and die.

Kids with Batten disease can develop normally for several years of life, but as the accumulation of waste continues, symptoms become sudden. The disease ultimately takes away the children's ability to walk, speak, see, and even identify their loved ones. The majority of kids with Batten disease don't live past their teens.

What Causes Batten Disease?

Batten disease is a hereditary disorder that results from mutations in genes, which interfere with proper cellular functioning. The condition has an autosomal recessive mode of inheritance, such that the child inherits two copies of the faulty gene—one from each parent—before the disease sets in. Only one copy of the gene in the parents often has no manifestation, but it gives them a 25% probability of passing on the disorder to the offspring during each conception.

Symptoms of Batten Disease

Batten disease varies in how it affects each child, but symptoms may include:

• Seizures and epilepsy
• Loss of vision to complete blindness
• Speech and language problems
• Loss of motor function, leading to an inability to walk and eventual paralysis
• Cognitive impairment and loss of memory
• Changes in personality and psychiatric disturbances such as aggression or anxiety
• Dementia-like features at an early age
• Extrapyramidal features such as tremor, rigidity, and restlessness

The rate at which the symptoms develop differs from child to child, but the ultimate outcome is always tragic—complete neurological degeneration and premature death.

How Common Is Batten Disease?

Batten disease is very rare, with a prevalence of about 3 cases per 100,000 births in the United States. In the UK, only about 40 children are being treated for the condition, and one to six children are born with Batten disease each year.

Treatment Options: Is There Any Hope?

Currently, there is no cure for Batten disease. Nevertheless, medical research has produced some encouraging treatments:

Enzyme Replacement Therapy (ERT): Brineura, a medication by BioMarin, is the sole treatment approved for one type of Batten disease (CLN2). It slows disease progression but neither halts nor reverses damage.

Gene Therapy: Scientists are exploring gene therapy methods that will replace faulty genes with normal genes. This is still in the experimental phase.

Supportive Care: Because there is no certain cure, treatment is largely symptom management by way of anti-seizure medications, physical therapy, speech therapy, and mobility and communication assistive devices.

Challenge To Access to Treatment

One of the big issues with Batten disease treatment is accessibility. The sole drug available, Brineura, has a colossal price tag—at more than £500,000 per patient annually. This has made temporary access in the UK possible after an agreement under a Managed Access Agreement (MAA) between the National Health Service (NHS) and BioMarin, but the agreement is to lapse in May unless fresh negotiations are successful.

The expense of treatment is so high that it generates ethical and economic arguments over whether life-prolonging treatments for ultra-rare conditions should be provided despite the cost. For families with Batten disease, the prospect of losing access to this life-prolonging treatment is horrific.

Unfortunately, the prognosis for children with Batten disease is poor. The disease is always terminal, with life expectancy differing based on the particular type of Batten disease. The majority of children diagnosed with the disorder do not live beyond their teenage years.

As the disease advances, children lose the capacity to speak, move, and identify people they love. Ultimately, organ failure occurs, resulting in premature death. Though research continues in hopes of developing improved treatments and eventually a cure, the reality for Batten disease families at present is heartbreakingly challenging.

Batten disease is a heartless and unrelenting disease that robs children of their potential and families of their dreams. With the progress of science, there remains hope that gene therapy and other new treatments could one day provide a cure. Until then, awareness, advocating for affordable treatment, and funding research are imperative steps in the battle against Batten disease.

Until then, suffering families and doctors must endure the hardship of this lethal disorder, awaiting a turning point that will revolutionize the destiny of children afflicted with Batten disease.

Every year on 25 April, World Malaria Day is observed to raise awareness about one of the deadliest yet preventable diseases. As per the World Health Organization (WHO), there were 263 million malaria cases and 5,97,000 malaria deaths across 83 countries in 2023. The WHO African Region carries a disproportionately high share of the global malaria burden. As per the numbers, this Region was home to 94% of the malaria cases, accounting for 246 million and 95% of malaria deaths. Children under 5 accounted for about 75% of all malaria deaths in the Region.

Ahead of World Malaria Day, WHO also called for renewed efforts at all levels - from global policy to community action to accelerate progress towards eliminating malaria.

World Malaria Day 2025 Theme

This year, WHO has joined the RBM Partnership to End Malaria and other partners in promoting: "Malaria Ends With US: Reinvest, Reimagine, Reignite". This is a grassroot campaign that aims to re-energize efforts at all levels, from global policy to community action, to accelerate progress towards malaria elimination.

World Malaria Day History

World Malaria Day was first celebrated internationally in 2008, building upon the earlier "Africa Malaria Day", which had been observed b African countries since 2001. The date, April 25, was established by WHO in 2007 during the World Health Assembly. In 2007, it was the 60th session of the World Health Assembly where the proposal to rename Africa Malaria Day to World Malaria Day was made to acknowledge the global presence of malaria.

World Malaria Day Significance

The day has a strong significance in healthcare as it brings attention to the disease that still continues to take so many lives, especially in low-income and tropical regions. It also serves as an important reminder to continue spreading awareness about the disease as well as promoting its prevention, treatment and continuous international cooperation to fight against it.

What Is Malaria?

The WHO describes malaria as a life-threatening disease spread to humans by some types of mosquitoes, mostly found in tropical countries. However, they are preventable and curable.

WHO notes: "Malaria is spread to people through the bites of some infected anopheles mosquitoes. Blood transfusion and contaminated needles may also transmit malaria. The first symptoms may be mild, similar to many febrile illnesses, and difficult to recognize as malaria. Left untreated, P. falciparum malaria can progress to severe illness and death within 24 hours.

There are 5 Plasmodium parasite species that cause malaria in humans, and 2 of these species – P. falciparum and P. vivax – pose the greatest threat. P. falciparum is the deadliest malaria parasite and the most prevalent on the African continent. P. vivax is the dominant malaria parasite in most countries outside of sub-Saharan Africa. The other malaria species which can infect humans are P. malariae, P. ovale and P. knowlesi."

What Are The Symptoms?

The early symptoms are fever, headache and chills, which can usually start within 10 to 15 days of getting bitten by an infected mosquito.

Some types of malaria can cause severe illness and death. Infants, children under 5 years, pregnant women, travellers and people with HIV or AIDS are at higher risk. Severe symptoms include:

• extreme tiredness and fatigue
• impaired consciousness
• multiple convulsions
• difficulty breathing
• dark or bloody urine
• jaundice (yellowing of the eyes and skin)
• abnormal bleeding

A new AI-powered blood test that can detect 12 types of cancer is to be tested on NHS patients. Using this test, experts would now be able to detect cancerous cells in people much before the symptoms appear. The trial, conducted on 8,000 patients, will analyse blood samples for tiny fragments of genetic material released by tumours.

The test called miONCO-Dx, was created using data from 20,000 patients. Initial tests have produced promising results, having shown that it can detect 12 of the most lethal and common cancers, including bowel cancer, at an early stage, with over 99% accuracy. With no other trial currently working in the same way, this is a world-leader and will support in placing Britain at the forefront of revolutionising healthcare. Notably, the UK government has awarded £2.4m to run the trial of the genetic test, which was developed by the University of Southampton and the biotech startup Xgenera.

How Does It Work?

The test was created by Xgenera, in collaboration with the University of Southampton. As little as 10 drops of blood are all that's needed to detect up to 12 common cancers. The test works by measuring the microRNA in a blood sample and using AI to identify if cancer is present and, if so, where it is located in the body.

What Cancers Will It Detect?

Lung, gastric, prostate, oesophageal, liver, bladder, ovarian, bowel, pancreatic and breast cancers–as well as bone and soft tissue sarcoma and a type of brain tumour. The Department of Health said the test was now ready for the "validation and verification" stage.

Professor Sir Stephen Powis, NHS England's national medical director, said: "This blood test has the potential to help us detect bowel cancer earlier and reduce the need for invasive tests, and the next step in this trial will now be vital in gathering further evidence on its effectiveness and how it could work in practice."

How Is A Blood Test Conducted?

A blood test is a simple medical procedure used to check various health conditions. Here's how it is typically conducted:

1. Preparation: Depending on the test, you may be asked to fast for 8–12 hours. The healthcare provider will explain any specific instructions.

2. Collection: You’ll usually be seated or lying down. A healthcare professional will tie a tourniquet around your upper arm to make the veins more visible. The inside of your elbow is the most common site for drawing blood.

3. Cleaning: The area is cleaned with an antiseptic to reduce the risk of infection.

4. Drawing Blood: A sterile needle is inserted into the vein, and blood is collected into one or more vials or tubes.

5. Post-collection: Once enough blood is collected, the needle is removed, and a cotton ball or bandage is applied to stop any bleeding.

6. Processing: The blood samples are then sent to a lab for analysis.

Novovax, the maker of the only protein-based COVID-19 vaccine available in the US announced that its shot is on track for full approval from the US Food and Drug Administration (FDA). It is an important development for the company. It has sent its stock soaring up to 21% on Wednesday morning for trading. It is said that this will also ease the fears of political interference that may have caused delay in the process.

Vaccine For Emergency Use Only

While the other mRNA vaccines from Pfizer and Moderna have received full FDA approval for specific age groups, Novovax's vaccine still awaits the approval. It is only authorized for emergency use.

The emergency use authorization or the EUA allows vaccines to be distributed during public health emergencies. However, once the emergency ends, the FDA can remove them from the market unless full approval is granted.

Why Did The Delay Happen?

The FDA originally planned to approve Novovax's vaccine by April 1. However, as per the inside sources, the process was paused at the direction of Dr Sara Brenner, the FDA's acting commissioner. The delay has also raised concerns, especially after Dr Peter Marks, the FDA's longtime vaccine chief, reportedly left his post due to disagreements with the Health Secretary Robert F Kennedy Jr, who is a known vaccine skeptic.

What Makes Him A Vaccine Skeptic?

In the past, RFK Jr. has worked closely with many anti-vaccine activists who work for his nonprofit group Children's Health Defense. While in his recent speech, he said that he has "never been anti-vax and have never told the public to avoid vaccination", his track record shows otherwise.

In a podcast interview, he said, "There is no vaccine that is safe and effective" and told FOX News that he still believes in the now long-debunked idea that vaccines can cause autism. In another 2021 podcast, he urged people to "resist" CDC guidelines on getting their kids vaccinated. "I see somebody on a hiking trail carrying a little baby and I say to him, better not get them vaccinated," he said.

His non-profit also led an anti-vax campaign sticker and he appeared next on the screen to a sticker that read: "If you are not an anti-vaxxer you are not paying attention," reports AP.

What Does The Vaccine Need For Approval?

The FDA recently asked Novavax to outline a plan to collect additional clinical data from people who have received the vaccine. Novavax says it is “engaging with the FDA expeditiously” and hopes to secure full approval as soon as possible. Full FDA approval is considered the gold standard, as it reflects a higher level of scrutiny and confidence in a product’s safety and effectiveness.

How Is This Vaccine Different From Others?

The COVID-19 vaccines that are currently available in the US teach the immune system to recognize the virus' spike protein, which is its outer coating. the Pfizer and Moderna's mRNA vaccine deliver genetic instructions that help the body create a temporary version of spike protein that trigger an immune response. In contrast, the Novovax's shot contains lab-grown copies of the spike protein itself, which are then combined with a substance that boosts the immune response.

This traditional approach—called a protein-based vaccine—has been used for decades in vaccines for diseases like hepatitis B and shingles. For people who are hesitant about mRNA vaccines, Novavax offers an alternative that uses a well-established method.