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.

Shocking to many, expected for more, the US Health Secretary Robert F Kennedy Jr on Monday removed every member of a scientific committee that advises the Centers for Disease Control and Prevention (CDC) on how to use vaccines. He has also pledged to replace them with his own picks.

This is the Advisory Committee on Immunization Practices (ACIP), from where 17 members have been removed. This step has been criticized by many physicians and public health groups.

It may come as a shock to many, but in all fairness, it was known that Kennedy was among the nation's leading anti-vaccine activists before he became the nation's top health official. While he has revealed that the members will be his own pick, he has not said who would he appoint. However, as per reports, this should convene in two week in Atlanta. It is also important to note that the entire roster of committee members were in fact, Biden appointees.

In an opinion piece published in the Wall Street Journal, Kennedy wrote: “Without removing the current members, the current Trump administration would not have been able to appoint a majority of new members until 2028. A clean sweep s needed to re-establish public confidence in vaccine science."

The reports say that the former panel members received an email on a late Monday afternoon that said that their services had been terminated without any reason. Many of whom had not served a full term.

While his steps are being criticized, they must not be seen as a shock as Kennedy had already taken the unusual step of changing COVID-19 recommendations without first consulting the committee.

As per Kennedy, the committee members had too many conflict of interests. However, as the US News reports, Dr Tom Frieden, who is the president and CEO of Resolve to Save Lives and former director of CDC said that Kennedy's actions were based on false conflict of interest claims and that they set a "dangerous and unprecedented action that makes our families less safe". As this step can significantly reduce vaccine access to many people.

“Make no mistake: Politicizing the ACIP as Secretary Kennedy is doing will undermine public trust under the guise of improving it,” he said in a statement. “We’ll look back at this as a grave mistake that sacrificed decades of scientific rigor, undermined public trust, and opened the door for fringe theories rather than facts.”

ALSO READ: What Are The Key Takeaways From RFK Jr.'s CDC Press Conference On Autism Report?

RFK Jr.'s Past Remarks On Vaccines

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. He told the FOX News that "autism comes from vaccine". However, vaccination has indeed saved more lives.

After taking the charge of the office, Dr Peter Marks, one of the top US Food and Drug Administration (FDA)'s leaders and scientist in vaccine regulation had also been ousted. Several sources have also reported that his resignation was not voluntary, in fact he was reportedly told to 'resign or get fired'.

Stay Updated On CDC and RFK Jr. Here

The Unites States reported 122 new cases of measles last week, which has brought the national total to 1,168 in 2025. This data is released by the Centers for Disease Control and Prevention (CDC). While the outbreaks in Pennsylvania and Michigan have officially ended, others across North America continue to unfold, including in parts of Texas, New Mexico, and Canada.

Resurgence of a Vaccine-preventable Disease

Measles is caused by a highly contagious airborne virus that spreads through coughing, sneezing, or even breathing near an infected person. It had been declared eliminated from the U.S. in 2000 thanks to widespread vaccination efforts. However, recent declines in vaccination rates and increased international travel have led to a resurgence. The disease can lead to severe complications such as pneumonia, encephalitis (brain swelling), and death, especially in unvaccinated individuals.

The best defense against measles remains the MMR (measles, mumps, rubella) vaccine, typically administered in two doses during early childhood. Yet, the U.S. has seen a growing number of vaccine exemptions, which health experts say undermine community immunity.

Texas Update

Texas experienced the country’s largest outbreak this year, with 742 cases reported across 35 counties. Most were concentrated in Gaines County, where a close-knit, undervaccinated Mennonite community accounted for more than half of the state’s total cases. Two unvaccinated children — aged six and eight — died in separate incidents earlier this year.

As of this week, fewer than 10 individuals in Texas are estimated to still be infectious. State health officials will now provide updates weekly instead of daily, suggesting the worst of the outbreak may be over.

Active Outbreaks In Other States

As per CDC if a state reports three or more related cases, it would be defined as an outbreak:

• New Mexico has reported 81 total cases and one death. Most are in Lea County.
• Oklahoma has 15 confirmed and three probable cases, though locations remain undisclosed.
• Colorado has logged 12 cases, including a seven-case outbreak tied to a Turkish Airlines flight in May.
• Illinois reports eight cases in its southern region and two in Cook County.
• Kansas has 71 cases, mostly in Gray County.
• Montana, facing its first outbreak in 35 years, has 17 cases.
• North Dakota now has 34 cases, all among unvaccinated individuals.
• Ohio has reported 34 cases in two separate outbreaks.
• Canada and Mexico See Major Outbreaks Too

Canada’s Ontario province has seen the longest outbreak in North America, with 2,009 cases and one death in a baby with congenital measles. Alberta has 761 cases. In Mexico, the state of Chihuahua reported 1,940 cases and four deaths.

Symptoms and Treatment

Measles symptoms start with high fever, cough, runny nose, and red eyes, followed by a spreading rash. Complications can be severe, especially for young children and immunocompromised individuals. There is no specific antiviral treatment; care typically focuses on symptom management and avoiding further spread.

As the summer travel season peaks, the Centers for Disease Control and Prevention (CDC) has issued an important advisory to travelers about the re-emergence of measles in the US, specifically highlighting the risk of contracting the highly contagious disease at airports and other crowded transit points. Once being declared eradicated from the United States in 2000, measles has has made a unsettling comeback, fueled primarily by declining vaccination rates and global travel. With more than 1,100 reported cases across the country this year alone, the CDC's warning is a harsh reminder that this preventable illness is a very real and present danger—particularly in areas thick with travelers.

For generations, measles was a thing of the past in the U.S. due to widespread vaccination campaigns. The disease was almost eradicated over the last two decades, with only sporadic imported cases and small outbreaks occurring. Recent trends, though, have turned this tide of progress around. This year's figure of 1,157 cases already exceeds numbers from earlier years, indicating a public health problem not witnessed since 2019, when a large-scale outbreak rocked communities.

Much of this increase stems from a multistate outbreak concentrated in West Texas, but the risk is far from localized. The CDC highlights that the virus is highly contagious and can spread rapidly in places like airports, train stations, planes, crowded events, and tourist attractions—anywhere large numbers of people converge, particularly if some are unvaccinated or susceptible.

Why Airports and Travel Hubs Are High-Risk Zones?

Airports stand out for their ability to transmit disease. They are gateways to and from around the world with tightly packed crowds, frequently crowded waiting rooms, and intimate mingling among travelers from all parts of the globe—some from nations where measles remains endemic. The CDC travel notice emphasizes that travelers can catch measles while in these locations, then return home with the virus and spread it further.

The extreme infectiousness of measles makes it so that casual exposure to an infected individual or contact with contaminated surfaces can result in infection. Fever, cough, rash, and serious complications such as seizures and blindness are all symptoms that make it a dangerous virus, particularly for infants, pregnant women, and the immunocompromised.

Why Is Measles Surging?

The resurgence is largely due to vaccine hesitancy and disinformation that have caused some parents to skip or postpone childhood vaccinations. The implications are evident: areas of under-immunized communities are fertile breeding grounds for measles outbreaks. Anti-vaccine attitudes, in some cases spurred on by misinformation and suspicion of medical institutions, have been a prime force behind the current resurgence.

Adding to this problem is the amount of travel anticipated this summer. As millions of American travelers flood airports, trains, and vacation spots, the risk of transmission increases, particularly in areas with lower vaccination levels.

Vaccine Guidance and Precautions for Travelers

The CDC advises all international travelers to make sure they are up to date with their measles vaccination, including two doses of the measles-mumps-rubella (MMR) vaccine at least 28 days apart. Infants 6 to 11 months old traveling internationally should also receive an additional dose of MMR vaccine prior to travel.

Those born prior to 1957 are presumed to have had natural immunity from childhood exposure and thus are not recommended to be vaccinated by the CDC or Texas public health officials except in the case of healthcare personnel who work in outbreak areas, for whom two doses should be administered regardless of year of birth.

Some individuals, like pregnant women, infants younger than six months, and individuals with severely compromised immune systems, are not eligible for the vaccine. These persons should speak with their physicians regarding their individual risk and possible preventive action, like immunoglobulin therapy, that will provide temporary protection once exposed.

How to Stay Safe While Traveling?

Experts recommend that travelers take a proactive role by getting their immunity status checked sufficiently in advance of their travel—ideally six weeks or more ahead. This can include checking vaccination history, laboratory tests, or evidence of previous infection. Suspected exposure to measles calls for immediate medical consultation, since early administration of MMR vaccine or immunoglobulin can cut down on disease severity.

Along with vaccination, routine preventive precautions are important: frequent washing of hands, avoidance of contact with the sick, remaining in well-ventilated places, and use of masks when required, particularly for those with high risks of developing complications.

Travelers need to prepare as well by bringing along medications that are necessary and learning about healthcare facilities in the destinations. Checking the CDC's travel health notices may offer current information on risks and vaccine requirements for countries.

The recent measles outbreak is a cautionary example of the vulnerability of disease elimination efforts. Even as vaccination campaigns have long kept measles at bay, recent slippages risk undoing decades of gains. The warnings from the CDC occur at a time when wider concerns exist regarding vaccine misinformation and waning public trust in immunizations.

The consequences are dire. Measles is not a harmless childhood disease; it can result in severe complications, hospitalization, and even death. Already this year, at least two children and one adult have died from measles-related complications in the United States, and these figures may increase as travel continues and outbreaks multiply.

Travelers need to understand that immunity is the best defense against measles. Howsoever healthy your living style, germs do not distinguish between people—germs infect the unimmunized, the weak, and those who come in close proximity to carriers equally.