Latest news with #MLD
Daily Mail
a day ago
- Health
- Daily Mail
Revealed: The most eye-wateringly pricey drugs that the NHS is paying some £3million a dose for...while dementia and cancer patients are denied cheaper treatments
NHS bosses are paying millions of pounds for just a single dose of medication to treat some of the most devastating health conditions in the world. The drugs—some of which cost just shy of £3million per patient—can revolutionise and even save the lives of a handful of people born with rare illnesses each year. But it comes as Government officials have deemed cheaper treatments for far more common conditions like dementia and breast cancer as not worth the money. Libmeldy is widely cited as the most expensive drug available on the NHS, coming in at an eye-watering £2.875million per dose. It's used to treat babies with a fatal genetic disease called metachromatic leukodystrophy (MLD). While born seemingly perfectly healthy, children with MLD have a faulty gene that leads to a destructive build-up of fat around their nerves. Over time, this severely damages their brain and nervous system, with parents forced to watch helplessly as their child gradually stops walking, talking and then eating. Roughly four babies are born with MLD every year in the UK, and have a life expectancy of just five to eight years. But Libmeldy, a one-off treatment administered as an IV drip in hospital, uses a specially designed harmless virus to alter a patients' cells and remove the faulty gene that drives MLD. It's a bespoke treatment that uses a patient's individual cells, meaning a dose is unique to each individual. Another treatment that works in a similar way is Hemgenix, which MailOnline revealed was administered for the first time by the NHS earlier week. The drug —which costs an estimated £2.6million per patient—is also a gene therapy, delivered as one-off IV drip. It is the only treatment of its kind for haemophilia B, a bleeding disorder where the body doesn't make enough—or any—of a vital protein critical to clotting. Clotting stops wounds from bleeding, so those with the disorder risk suffering severe and even life-threatening blood loss from even minor injuries. Patients also run the risk of what are called 'spontaneous bleeds', which can be triggered without a direct injury and even prove deadly if they occur in a vital organ. Prior to Hemgenix, all haemophilia B patients needed regular weekly injections of an artificial clotting agent to keep their risk of catastrophic injury to a minimum. This meant many patients were effectively tethered to their scheduled injections, unable to live their lives without worry and anxiety about every potential nick and scrape. Similar to Libmeldy, Hemgenix works by replacing a patient's defective gene—which is incapable of producing the clotting protein—with one that can, eliminating the need for regular injections. Studies suggest the protective effect lasts for at least three years, but the hope is that it could work for even longer. There are approximately 2,000 people with haemophilia B in the UK. Yet, only around 260 with 'moderately severe or severe haemophilia B' are currently eligible for Hemgenix on the NHS. This puts the potential total bill to the taxpayer at roughly £676million. While a cost of £2.6million per patient may seem prohibitive, medics claim the treating patients this way actually saves the NHS money in the long term. The lifetime cost of providing a patient the alternative weekly clotting injections has been estimated to be £8million. This sum doesn't include the cost of life-saving interventions and surgeries haemophilia B patients may also need. Another multi-million drug approved for use on the NHS is Zolgensma, which comes in at £1.8million per dose. It's designed to help babies with spinal muscular atrophy (SMA), a genetic disease that typically kills within two years if left untreated. Around 56 babies are born with SMA each year, with the condition caused by a defective gene that plays a critical role in allowing nerves in the spinal cord to control muscles movement. SMA causes muscles to waste and gets worse over time, making it difficult for patients to breathe, move and eat. Nine in 10 of those who have the most severe form of the disease, known as type 1, die by the age of two if they do not receive treatment. Zolgensma is given as one off infusion—that like the previously discussed gene therapies—also fixes the faulty gene that drives SMA. Gene therapies are incredibly expensive medications due to the intense design and manufacturing process that goes into their creation. Additionally, because many of them benefit rare health conditions with small patient populations there is an argument that companies need to charge high prices to recoup costs given the drug may only be used a handful of times per year. But exactly how much the NHS pays for these drugs isn't clear. While the companies which make the drugs do charge millions for them, the health service often acquires the medications at an undisclosed discount, so the actual cost is likely to be less than it first appears. Which drugs get funded on the NHS in England and Wales is determined by the NHS spending watchdog, The National Institute for Health and Care Excellence (NICE). In making its decisions, NICE considers a multitude of factors. These include a drug's clinical impact—how much it improves or alleviates a patient's condition—as well as risk of side effects, practical issues like specialist storage, if there are alternatives, and, critically, how much it costs. It then judges if funding the drug will overall provides value for taxpayer money. This is often summarised using a metric called a quality-adjusted life year (QALY), essentially how much it costs to give a patient a healthy year of life. Lecanemab (pictured) and donanemab are currently only available to Brits who can afford to pay around £60,000 privately every year at select clinics Generally, if a drug costs about £20,000 to £30,000 per QALY NICE usually assesses it as a good use of taxpayer funding. This isn't a hard limit. The watchdog can rule drugs that are expensive and only work for a limited time, like end-of-life medications as worth the cost, recognising small amounts of time can be worth a lot to patients and their families. However, this process isn't an an exact science and can attract controversy. For example, the breast cancer drug Enhertu, described as a lifeline by campaigners, has been deemed too expensive by NICE for what it does. However, Scotland's equivalent of NICE—the Scottish Medicines Consortium—has deemed the £10,000 per patient per month drug as value for money. Research suggests Enhertu extends the lives of patients with one of the hardest to treat forms of breast cancer, buying them an extra year or more of life. Given as an infusion, it helps patients with an aggressive and fast-growing type of the cancer called HER2-positive. NICE previously accused the firm behind the drug, AstraZeneca, of refusing to 'offer a fair price'. About 57,000 cases of breast cancer are diagnosed in the UK each year with HER2-positive cancers accounting for roughly one in five of these, some 11,500. Other drugs that have attracted controversy after being rejected by NICE are donanemab and lecanemab. Both medications are designed slow down the early stages of Alzheimer's disease, the leading cause of dementia. The drugs bind to amyloid, a protein which builds up in the brains of people living with Alzheimer's, helping to clear out the substance and slowing cognitive decline. However—in its most recent ruling—NICE said while the treatments worked they only delayed the progression from mild to moderate Alzheimer's by four to six months. As such, the body ruled the medications cannot be provided on the NHS because they are not good value for money and 'only provide modest benefits at best'. Charities described the decision as 'disappointing' and a 'painful setback' for patients, while the firms Lilly, which makes donanemab, and Eisai, which makes lecanemab, said they would appeal the ruling. Alzheimer's is the most common cause of dementia in the UK with 944,000 Britons estimated to be living with the memory robbing disorder. NHS England published a briefing paper last year suggesting the cost of bringing the drugs to the health service could be £500 million to £1 billion per year. While multi-million purchases of single dose drugs using taxpayer funds—as people with far more common condition are denied cheaper medications—may raise eyebrows, the cost is worth putting into context. The NHS purchasing a one off dose of a £2.875million to save a child who would otherwise die, costs roughly 4p per person in the UK. This sum is about half of £5million the NHS in England spends on dishing out the over-the-counter painkiller paracetamol every month, despite the health service banning GPs from prescribing such cheap drugs to patients in 2018.
Tatler Asia
30-05-2025
- Health
- Tatler Asia
Why everyone is talking about lymphatic drainage—and what it really does
What it actually does (and doesn't do) Despite the influencer hype, lymphatic drainage does not melt fat or deliver permanent slimming effects. What it does do is reduce water retention and localised swelling, particularly after long-haul flights, illness or salty meals. Manual lymphatic drainage (MLD)—a technique performed by a trained therapist—involves light, rhythmic strokes that guide lymph toward the body's drainage points, such as the collarbones or groin. Studies support its efficacy in reducing post-surgical swelling, alleviating chronic inflammation, and improving circulation in sedentary individuals. However, its 'detoxifying' effects are often misunderstood. The liver and kidneys are responsible for detoxification, not the lymphatic system. That said, lymphatic drainage supports the body's natural waste-removal processes by helping prevent stagnation and puffiness. What is not lymphatic drainage Above Gua sha, although often touted as a lymphatic drainage treatment, is a technique that improves blood flow and reduces inflammation. Not everything labelled as lymphatic drainage delivers on its promise. Gua sha, for instance, is rooted in Traditional Chinese Medicine and is primarily designed to enhance blood flow and relieve muscle tension—not necessarily lymph flow. While its gentle motions may look similar to MLD, unless the practitioner is trained in lymphatic anatomy, the effects remain mostly superficial. The same goes for foam rolling, deep tissue massages, and aggressive facial-sculpting devices, which often fall under the same marketing umbrella. These may improve circulation or ease muscle tension, but they don't always support lymphatic function. In fact, excessive pressure can compress lymph vessels, making matters worse. True lymphatic drainage relies on feather-light, directional techniques aligned with the body's lymphatic pathways. If your treatment leaves you sore or bruised, it wasn't done correctly. Why lymphatic drainage is trending now Lymphatic drainage taps into a bigger shift in wellness culture: the growing appeal of restorative rituals over punishing routines. Unlike high-intensity workouts or juice fasts, it offers gentle results with minimal effort—just time, access and a skilled practitioner. Social media has propelled its popularity, with up-close videos of facial massages and 'post-drainage glow-ups' reinforcing its appeal. Influencers often link it to aesthetic buzzwords like 'sculpted', 'de-puffed' and 'snatched jawline', though the biological mechanisms are more circulatory than structural. Luxury clinics in Hong Kong, Manila and Singapore now feature lymphatic drainage alongside LED facials, fascia tools and body-sculpting devices. Some treatments are manual, while others use pneumatic compression suits or radiofrequency. The real draw? A feeling of recalibration. In fast-paced cities where burnout is common, the idea of inner rebalancing sells—and lymphatic drainage delivers just that. Book only with trained experts When performed correctly, lymphatic drainage is generally low-risk. However, it's not suitable for everyone. Individuals with acute infections, heart failure or kidney issues should avoid the treatment unless cleared by a physician. Technique matters. TikTok tutorials and at-home tools can't replace professional training in anatomy or clinical contraindications. As wellness continues shifting from performance to restoration, demand for treatments that regulate rather than push the body will likely increase. Once a niche therapy, lymphatic drainage is now central to self-care conversations, sitting alongside fascia release and high-tech skin treatments. Will it transform your body overnight? No. But if what you're seeking is subtle relief, less puffiness and a deeper sense of ease, then it's worth the attention it's getting.
Yahoo
13-05-2025
- Health
- Yahoo
Top 10 most expensive prescription drugs in the US by price and by sales volume
On the heels of President Donald Trump's Monday announcement of an executive order that will slash prescription drug prices in the U.S., the spotlight is on current costs and how much Americans could save. The president's order calls for "most favored nations drug pricing" — which means "the lowest price paid for a drug in other developed countries, that is the price that Americans will pay," he said. "Some prescription drug and pharmaceutical prices will be reduced almost immediately by 50 to 80 to 90%," Trump said. President Trump Takes On 'Big Pharma' By Signing Executive Order To Lower Drug Prices Katy Dubinsky, a New York pharmacist and founder and CEO of Vitalize, applauded the move to reduce prescription prices, noting that Trump's order tackles a long-standing problem. "But this will not be simple to accomplish," she told Fox News Digital. Read On The Fox News App "The executive order doesn't reduce costs immediately," she said. "It directs government agencies to start drafting the rules, which may take months." Here are the five most expensive prescription drugs in the U.S. by price — followed by five by volume. Dubinsky detailed some of the most expensive prescription drugs in the country today and what conditions they treat. 1. Lenmeldy (atidarsagene autotemcel) by Orchard Therapeutics – $4.25 million This medication is used to treat metachromatic leukodystrophy (MLD), a rare genetic disorder that damages the nervous system, Dubinsky said. "It is given once and is supposed to stop or slow down the disease in young kids," she noted. Top 10 'Allergy Capitals' Of The Us, Plus 4 Tips To Manage Symptoms 2. Hemgenix (etranacogene dezaparvovec-drlb) by CSL Behring – $3.5 million This medication is prescribed for people with hemophilia B, a bleeding disorder. "This one-time treatment helps the body make its own clotting factor, so patients don't need regular infusions," said Dubinsky. 3. Elevidys (delandistrogene moxeparvovec-rokl) by Sarepta Therapeutics – $3.2 million This prescription medication, intended for young boys, treats Duchenne muscular dystrophy (DMD), a condition that weakens muscles over time. "It aims to slow down how fast the disease progresses," Dubinsky said. 4. Skysona (elivaldogene autotemcel) by Bluebird Bio – $3 million "This medication is used for cerebral adrenoleukodystrophy (CALD), a serious brain disease in boys," said Dubinsky. "This therapy tries to slow the damage before symptoms get worse." 5. Zynteglo (betibeglogene autotemcel) by Bluebird Bio – $2.8 million Zynteglo is for beta-thalassemia, a blood condition that usually requires regular transfusions. "This gene therapy can help patients make healthy red blood cells on their own and reduce how often they need treatment," said Dubinsky. John Stanford, executive director of Incubate, a Washington-based coalition of early-stage life-science investors, shared his thoughts on the top five most expensive drugs by sales volume. "Typically, when the government is focused on the most expensive drugs, they're focused on the metric based on sales volume rather than, for instance, a rare disease therapy with a high list price but smaller patient pool," he told Fox News Digital. "Often, officials are focused on total drug spending by Medicare or other government programs." 1. Keytruda (pembrolizumab) by Merck — $25 billion revenue (2023) Keytruda is an immunotherapy medication used to treat a variety of cancers, including melanoma, non-small cell lung cancer, liver cancer and others. "Keytruda has become Merck's crown jewel, helping the company expand its cancer treatment portfolio with more than 1,000 active clinical trials," Stanford told Fox News Digital. Terminal Colon Cancer Patient Saved By Breakthrough Treatment 2. Eliquis (apixaban) by Bristol Myers Squibb and Pfizer — $18.95 billion Eliquis (apixaban) is an "anchor drug" for both BMS and Pfizer, according to Stanford. Apixaban is prescribed to prevent the formation of blood clots and to treat deep vein thrombosis and pulmonary embolism (a blood clot in the lungs). 3. Ozempic (semaglutide) by Novo Nordisk — $13.93 billion Prescribed for type 2 diabetes, the semaglutide medication Ozempic has become widely popular for its weight-loss effects and other health benefits. "Ozempic's sales are powering Novo Nordisk's broader foray into GLP-1s for obesity, heart disease and liver conditions — all areas with high development costs and uncertain scientific outcomes," Stanford told Fox News Digital. "The money has gone toward scaling up production to meet demand for GLP-1s and avoid supply shortages." 4. Humira (AbbVie) — $14.4 billion (U.S. 2023 revenue) "Humira has been one of the highest-grossing drugs in history, generating over $200 billion during its exclusivity period," Stanford said. The injectable medication, which contains the active ingredient adalimumab, is used to treat rheumatoid arthritis and other inflammatory conditions. Click Here To Sign Up For Our Health Newsletter 5. Biktarvy by Gilead — $11.85 billion Biktarvy is an HIV treatment that includes the three ingredients bictegravir, emtricitabine and tenofovir alafenamide. "Biktarvy isn't just a leading HIV treatment — it's the financial backbone for Gilead's move into cancer research," Stanford said. Dr. Jacob Glanville, CEO of Centivax, a San Francisco biotechnology company, said vaccines and most generic drugs would not likely be changed by the executive order. "Most vaccines that Americans take cost less than a hundred dollars, while generic drugs are often less than a dollar a pill," he told Fox News Digital. What would be affected, Glanville predicted, are newer brand-name drugs still under IP exclusivity, antibody therapies, cellular therapies, gene therapies and personalized cancer vaccines. "Some of these are excruciatingly expensive — $100,000 to $500,000 for a treatment course for a patient. However, they are also often the most effective treatments for certain cancers, autoimmune disorders or rare diseases." The pharmaceutical industry might argue that lowering the prices on these medicines will result in a "dramatic reduction of investment" in creating such breakthroughs, said Glanville. The industry may also argue that these medicines eventually become generic — at which point the prices drop, according to the expert. For more Health articles, visit "From a patient's perspective, the price of medical care in the United States is unsustainable, and extremely expensive medicine is part of that," he said. But "the insurance system and the hospital business also contribute." "If the prices of new medicines are capped, then effort should be made to reduce the cost of clinical trials and drug GMP manufacturing. Otherwise, we will lose a lot of innovation." Greg Norman of Fox News Digital contributed article source: Top 10 most expensive prescription drugs in the US by price and by sales volume
Yahoo
10-05-2025
- Health
- Yahoo
Minnesota newborns to be screened for Metachromatic leukodystrophy (MLD)
The Brief The Minnesota Department of Health announced babies born in the state will be routinely screened for metachromatic leukodystrophy (MLD). Officials said screenings for the disease will likely start in 2026. MLD symptoms include patients not reaching developmental milestones, experiencing regression of skills, seizures and sometimes death. MINNESOTA (FOX 9) - Babies born in Minnesota will start to be routinely screened for metachromatic leukodystrophy (MLD), the state's health department announced. Officials say MLD will likely be added to the list of conditions Minnesota newborns are routinely screened for in 2026, based on the Newborn Screening Advisory Committee's recommendation. READ MORE: MN documentary chronicles groundbreaking treatment for rare genetic disease Big picture view MLD is a kind of lysosomal disease that is caused by an enzyme deficiency leading to a buildup of fats known as sulfatides. Health officials say the buildup causes brain and nervous system damage, which can lead to those diagnosed with MLD not reaching developmental milestones, experiencing regression of skills, seizures and sometimes death. There is no cure, but early intervention can slow progression of the disease and help manage symptoms. The Minnesota Department of Health said gene therapy "offers a promising approach" to treatment. Such a case was the subject of the documentary "Sequencing Hope", which followed an Alabama family's journey to M Health Fairview Masonic Children's Hospital in Minnesota for their daughter's treatment. Local perspective The Minnesota Department of Health (MDH) said the start date for MLD screening in Minnesota will likely be in 2026, with an exact date being determined and shared when the process unfolds. State officials add that Minnesota "has one of the most comprehensive newborn screening programs in the country" and point to screening for phenylketonuria (PKU) screening that started in 1964. Since then, all babies born in the state have had a blood sample sent to the Minnesota Department of Health for newborn screening unless parents opted out. Minnesota has since expanded its newborn screening panel to detect more than 60 conditions, allowing affected children to get the care they need as quickly as possible, according to the MDH. More information on the state's newborn screening program can be found here. The Source This story used information from a Minnesota Department of Health news release and past FOX 9 reporting.
Yahoo
17-04-2025
- Health
- Yahoo
HHS eliminates advisory committee on newborn screening ahead of vote on rare disorders
Health and Human Services Secretary Robert F. Kennedy Jr. has pledged in office to make Americans healthier, with a specific focus on reducing health burdens among children. But his department this month quietly eliminated an advisory committee on genetic disorders in newborns and kids. For the last 15 years, the central role of the Advisory Committee on Heritable Disorders in Newborns and Children was to make recommendations to the health and human services secretary about which conditions to include on a universal screening panel for newborns. Though Kennedy has been focused on identifying the origins of more pervasive childhood diseases like autism, asthma and obesity, rare diseases are collectively a large public health concern. Around 15 million children in the United States have rare diseases, most of which are genetic. Newborn screenings identify around 14,000 babies every year who have potentially life-threatening or life-altering conditions, according to the National Organization for Rare Disorders (NORD). Getting a diagnosis around the time of a child's birth gives parents a valuable window to seek treatment, which is usually most effective before symptoms set in. It's up to states to decide which conditions to test for, but most follow the federal government's Recommended Uniform Screening Panel, which suggests looking for 38 conditions, including cystic fibrosis and Pompe disease, a disorder that causes muscle weakness. The screening panel is largely shaped by recommendations from the advisory committee's volunteer scientists and medical experts. The committee has 'gone a long way in helping to ensure that newborns across the country, regardless of where they're born, are screened for these certain conditions,' said Allison Herrity, a senior policy analyst at NORD. According to an internal HHS email reviewed by NBC News, the committee was terminated, without explanation, on April 3. It had been scheduled to meet next month to discuss adding two conditions to the RUSP: metachromatic leukodystrophy (MLD) and Duchenne muscular dystrophy (DMD). Herrity said there had been an expectation that one or both conditions would be added. 'To have that wither on the vine because the committee has been dissolved is just sad and frightening,' said Dr. Shawn McCandless, head of genetics and metabolism at Colorado Children's Hospital, who has served on the committee in the past. A spokesperson for the Department of Health and Human Services said the department is complying with President Donald Trump's executive order to reduce the size of the federal government. NORD estimates that up to 450 babies are born with MLD or DMD every year. DMD, a genetic disorder that affects mostly boys, causes muscles to degenerate and weaken over time. Patients usually must use wheelchairs and die of respiratory or heart failure. MLD causes a decline in mental and motor functions, meaning patients often lose the capacity to eat, speak or swallow. Many die from secondary infections. 'What characterizes both of them, as well as many of the conditions that we identify through newborn screening, is a period of a relatively healthy baby — which makes it harder to diagnose clinically, but also it's just that window of opportunity to give effective treatment,' McCandless said. Both disorders have treatments that may extend a child's life or improve its quality, one of the main factors that determines whether screening would be useful. The Food and Drug Administration approved a gene therapy (which modifies the genes causing a person's illness) for Duchenne muscular dystrophy in 2023 and for metachromatic leukodystrophy in 2024. But with the advisory committee gone, there is little chance the conditions will be added to the universal panel. Only two states, Illinois and New York, screen newborns for MLD. And two more states, Minnesota and Ohio, screen for DMD. Jennifer McNary, whose son Austin died of DMD in February, called the decision to terminate the committee just before the vote 'super disappointing.' 'It has been so long coming,' she said. 'It's been so many pilot studies and so much money spent.' McNary knows firsthand about the benefits of an early diagnosis. Austin was diagnosed at age 3, when he was already having trouble walking. He died at age 26. Her second son, Max, was diagnosed with DMD at birth, allowing him to receive physical therapy and steroids for his muscles earlier in the course of his disease. That early diagnosis and treatment likely improved his quality of life, McNary said. Max was better able to tolerate gene therapy and walked until he was 17, she said, whereas Austin stopped walking at age 10. At age 23, Max uses a wheelchair and needs help getting in and out of bed, but he lives semi-independently and goes out with friends, McNary said. Doctors and advocates say it is not just screenings for MLD or DMD that are at stake now — the future of universal screening is also in jeopardy. Not all states have the resources to review evidence that could support screening for new conditions, Herrity said. Advocates fear a return to a patchwork system in which states with more money or political will offer comprehensive screenings and others do not. McCandless said having an independent body make recommendations also reduces the odds that advocacy groups or pharmaceutical companies will have an outsize influence on which conditions are evaluated or prioritized. 'The committee has created a rational and scientific approach to adding conditions based on evidence, not just based on passionate advocacy,' he said. It has also helped re-examine whether screening recommendations reflect the latest scientific knowledge. Ten years ago, the committee helped change the federal recommendations for tyrosinemia type I, a metabolic disorder that can be treated with a pill, allowing some patients to live normally. At the time, states were missing the majority of cases by screening for tyrosine, a protein in the blood. The committee recognized the need to look for a different chemical, McCandless said, and the disorder is now widely picked up by screening. McCandless and a dozen other doctors who treat rare disorders sent a letter this month urging Kennedy to reinstate the advisory committee, saying it 'aligns with your vision for a healthier America.' But some advocates, like McNary, hope for even more. 'Can we improve the process a bit, make it easier to add conditions at the same speed at which we're getting approved drugs?' she said. 'Maybe we can turn this into a positive and the administration could get a win by making something better." This article was originally published on
