Girl, 3, who was 'wobbly' now facing 'heartbreaking' condition
Mylah Algaradi is three-years-old. She was diagnosed at birth with a rare and debilitating condition called Arginase deficiency, which is part of the urea cycle of disorders.
This severe metabolic disorder is now relentlessly attacking Mylah's health, causing her mobility to deteriorate day by day.
READ MORE: Live updates as huge fire breaks out on docks
READ MORE: Man punched in the face in daylight attack at Central Station
Although Mylah has been dealing with this condition since birth, she had been developing well and was meeting all her key milestones having followed a strict low protein diet under the care of Alder Hey Children's Hospital and Manchester Children's Metabolic Team.
However, earlier this year, she began struggling to walk and within a matter of weeks she lost the ability to walk or stand altogether. She is also now struggling to talk.
TO DONATE TO MYLAH'S FUNDRAISING CAMPAIGN PLEASE CLICK HERE
Her family say the rapid progression of Arginase deficiency on Mylah has been both shocking and harrowing, robbing her of the simple joys of childhood mobility such as running around a room with friends or playing in the garden.
Mylah's mum, Maria, 35, told the ECHO: "It is just absolutely heartbreaking. This disease usually progresses slowly but with Mylah it all happened so quickly and took us all by surprise.
"We noticed her being a bit wobbly on her feet a few months ago, but she had recently been on antibiotics after an operation so we thought it was that. But within weeks she was barely able to walk."
Mylah is also now finding it harder to talk, struggling to say what she wants to say, with her words now coming out more slowly. Her family said they no longer get the "easy chats and giggles" they once enjoyed with their little girl as this cruel illness and its effects continue to get worse.
Maria added: "It is such a struggle for her to get her words out. For the first few days she just cried because she couldn't say what she was trying to say. The songs we used to hear her sing, she can't manage the first word of them anymore.
"It is so hard watching her decline every day. Her behaviour is changing. She can't play like she used to."
Mylah is currently on the waiting list for a liver transplant under Leeds Children's Hospital, where she has been since January 2024. Her family believed there was enough time for the transplant to happen before any of the very serious symptoms would begin.
Mylah's uncle - had put himself forward as a liver donor and is a blood group match, however the full assessment for this process will take some time.
To add to the situation, the little girl has had to have her tonsils removed to eliminate the risk of tonsillitis after the transplant when she will be heavily immune suppressed.
The reality is that the vital treatment Mylah now desperately needs to slow the progression of her disease - and potentially reverse it- is not currently available on the NHS.
The cost of Pegzilarginase is an eye-watering £4,600 per vial, working out at nearly £56,000 for a 12-week course. Without this treatment, Mylah's condition will continue to deteriorate and could lead to irreversible damage.
Mylah's mum contacted the manufacturer of the drug to apply for compassionate access, but was denied, her doctor's efforts to get individual funding from Alder Hey were also unsuccessful.
Maria says setting up a crowdfunding campaign was a last resort but the family are out of options as they desperately try to find the cash needed for the 12-week course of the drugs for their cherished little girl.
A gofundme page was started for Mylah on July 26 and has already amassed an impressive £18,000 - however much more is required to raise the £56,000 needed for the treatment she requires.
Maria said: "People have been absolutely amazing. I keep crying every time my phone pings and someone else sends a donation, whether it is £5, £50 or £100. There are so many people who I don't know, who I have never met, who are being so generous."
If you are able to contribute to Mylah's fund, please do so here or search 'Help Mylah Get Treatment To Fight Arginase Deficiency' on gofundme.com.
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Yahoo
3 hours ago
- Yahoo
Quoin Pharmaceuticals Releases Fourth Episode in NETHERTON NOW Video Series Featuring International Patient Advocate Mandy Aldwin-Easton
New Episode Highlights the Painful, Often Misunderstood Reality of Living with Netherton Syndrome and the Urgent Need for Effective Treatments ASHBURN, Va., Aug. 12, 2025 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) ('Quoin' or the 'Company'), a late clinical-stage specialty pharmaceutical company focused on rare and orphan diseases, today announced the release of the fourth episode in its NETHERTON NOW video series. This installment features Mandy Aldwin-Easton, a Netherton Syndrome patient, and a widely recognized, passionate advocate for people living with rare diseases. Mandy serves as Medical and Communications Manager and Co-founder of the UK-based Ichthyosis Support Group and is President of the European Network for Ichthyosis. In the video, Mandy shares her personal journey living with Netherton Syndrome, from delayed diagnosis, severe, chronic pain, multiple hospitalizations to the profound long-term impact this disease has had on her life. 'One thing we don't talk about is how incredibly painful skin is when it doesn't work properly,' Mandy explains in the video. 'Imagine millions of paper cuts all over your body. It's just so highly unpredictable. The one thing anybody wants with a rare disease like Netherton Syndrome is hope—that better treatments are on the way.' Mandy's story underscores the severe physical and emotional toll of the disease. Despite having a highly supportive family and medical advocacy, she has experienced extreme flare-ups, prolonged hospitalizations, and a lack of targeted treatment options throughout her entire life. As an adult, she continues to navigate the complexities of a disease that many still fail to recognize as life-threatening. 'I couldn't go out because my skin was falling off me. It was so incredibly painful that it was just unbearable to walk,' she recalls. 'Even with hospital stays and treatments, no one really knew how to care for my skin.' 'Mandy has long been a pillar of the ichthyosis and Netherton Syndrome communities,' said Denise Carter, Co-Founder and Chief Operating Officer of Quoin Pharmaceuticals. 'Netherton Syndrome is not just a rare skin disease. It is a relentless and punishing condition marked by excruciating pain, debilitating flare-ups, recurrent infections, and emotional trauma. The toll on patients and their families is immense, not only physically and emotionally but also financially, requiring lifelong care, frequent hospitalizations, and ongoing interventions that place a heavy burden on families and healthcare systems. Beyond her personal resilience, Mandy's unwavering advocacy has made her a powerful voice for this underserved and often overlooked population. We are honored to feature her in our NETHERTON NOW series and remain steadfast in our mission to bring awareness and hope to those living with this devastating disease.' The release of this video comes as Quoin continues to advance its lead product candidate, QRX003, in multiple pivotal clinical trials for the treatment of Netherton Syndrome. Recently reported data from ongoing studies have shown highly encouraging improvements in key clinical outcomes. 'Mandy's story reflects the extraordinary strength and perseverance of patients who have endured a lifetime of suffering without access to an approved treatment,' said Dr. Michael Myers, Chief Executive Officer and Co-Founder of Quoin Pharmaceuticals. 'As we advance QRX003 through multiple late-stage pivotal trials, voices like Mandy's reinforce the urgency of our mission. Quoin is committed to delivering a meaningful treatment for the severely underserved Netherton Syndrome community and to driving forward research that addresses the significant unmet needs across a broader range of rare skin disorders.' The full video featuring Mandy Aldwin-Easton is available at: About QRX003QRX003 is a topical lotion formulated with a proprietary delivery technology that contains a broad-spectrum serine protease inhibitor, whose mechanism of action is intended to perform the function of a specific protein called LEKTI. The absence of LEKTI in Netherton patients leads to excessive skin shedding, resulting in a highly porous and compromised skin barrier. QRX003 is designed to promote a more normalized skin-shedding process and the formation of a stronger and more effective skin barrier. For more information about Quoin's current clinical trials, visit: About Quoin Pharmaceuticals Pharmaceuticals Ltd. is a late clinical-stage specialty pharmaceutical company focused on developing and commercializing therapeutic products that treat rare and orphan diseases. We are committed to addressing unmet medical needs for patients, their families, communities and care teams. Quoin's innovative pipeline comprises four products in development that collectively have the potential to target a broad number of rare and orphan indications, including Netherton Syndrome, Peeling Skin Syndrome, SAM Syndrome, Palmoplantar Keratoderma, Scleroderma, Microcystic Lymphatic Malformations, Venous Malformations, Angiofibroma and others. For more information, visit or LinkedIn for updates. Cautionary Note Regarding Forward-Looking StatementsThe Company cautions that statements in this press release that are not descriptions of historical facts are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by the use of words referencing future events or circumstances, such as 'expect,' 'intend,' 'hope,' 'plan,' 'potential,' 'anticipate,' 'look forward,' 'believe,' 'may,' and 'will,' among others. All statements that reflect the Company's expectations, assumptions, projections, beliefs, or opinions about the future, other than statements of historical fact, are forward-looking statements, including, without limitation, statements relating to: bringing awareness and hope to those living with Netherton Syndrome, continuing to advance QRX003 through multiple late-stage pivotal trials, delivering a meaningful treatment for the Netherton Syndrome community, driving forward research that addresses the significant unmet needs across a broader range of rare skin disorders and Quoin's products in development collectively having the potential to target a broad number of rare and orphan indications, including Netherton Syndrome, Peeling Skin Syndrome, SAM Syndrome, Palmoplantar Keratoderma, Scleroderma, , Microcystic Lymphatic Malformations, Venous Malformations, Angiofibroma and others. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These forward-looking statements are based upon the Company's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties including, but not limited to, the Company's ability to deliver a safe and effective treatment for Netherton Syndrome; the Company's ability to pursue its regulatory strategy; the Company's ability to obtain regulatory approvals for commercialization of product candidates or to comply with ongoing regulatory requirements; the Company's ability to complete clinical trials on time and achieve desired results and benefits as expected, the Company experiencing unanticipated or higher than expected clinical trial costs; and other factors discussed in the Company's Annual Report on Form 10-K for the year ended December 31, 2024 and in other filings the Company has made and may make with the SEC in the future. One should not place undue reliance on these forward-looking statements, which speak only as of the date on which they were made. The Company undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as may be required by law. For further information, contact:Quoin Pharmaceuticals Myers, Ph.D., CEOmmyers@ Investor Contact:PCG AdvisoryJeff Ramsonjramson@ 863-6341
Yahoo
3 hours ago
- Yahoo
Government partners with Mounjaro maker to tackle obesity
The Government has joined forces with the pharmaceutical giant that makes weight loss jab Mounjaro in a bid to tackle obesity. The move could see patients accessing care at pharmacies or by using online platforms. The £85 million programme from Eli Lilly and the Department for Science, Innovation and Technology (DSIT) has been designed to look at how obese patients can access weight management care more easily. This includes through community services, in pharmacies and online. Using these tools, eligible patients could be treated 'in a matter of months', according to Health Secretary Wes Streeting. Under the agreement, the Government will contribute up to £50 million in UK-wide investment. Meanwhile, Eli Lilly will back the programme with £35 million, and NHS organisations will be able to apply for a share of the funding. Successful applicants can then use the cash to run and assess a pilot to build evidence for efficient obesity care in the UK. Eligible patients could be able to access these innovative services by summer 2026, the Government said. Mr Streeting warned that obesity is one of the leading causes of ill health and costs the health service billions. 'Yet, we now have the science, technology and knowledge to help tackle the obesity epidemic, if we seize this opportunity,' he added. 'This collaboration will help patients living with obesity in a matter of months – through testing better access to weight loss services and treatments. 'In the long-term it will inform how we can better tackle one of the biggest modern day health challenges and, through our Plan for Change, create an NHS that is fit for the future.' Science and Technology Secretary Peter Kyle said new ways of accessing support 'could be transformational for people's quality of life, and for society'. They could free obese people from 'ill-health that holds them back in daily life – while reducing the strain on our NHS', he added. NHS England national medical director Dr Claire Fuller said: 'Obesity is one of the biggest public health challenges we face and costs the NHS billions of pounds every year, so this collaboration between government and industry to drive new ways of supporting people with obesity could make a huge difference. 'The NHS is already developing and rolling out a range of ways to help people to manage their weight and live healthier lives, with patients benefiting from wraparound care from local weight management teams and via online support services, but we are excited by the potential of this collaboration to accelerate efforts to tackle obesity as the NHS moves from treatment to prevention as part of the 10-Year Health Plan.' Professor Rachel Batterham, senior vice president for international medical affairs at Lilly said: 'The launch of this programme marks an important milestone in advancing new models of care for obesity.' Henry Gregg, chief executive of the National Pharmacy Association, welcomed the announcement but warned that funding should cover costs for pharmacies. 'This is a vital step forward in addressing one of the most pressing health challenges facing our country,' he said. 'It's important that any funding made available is sufficient to cover the costs involved for pharmacies to provide wraparound care and support. 'Community pharmacies already support over 1.4 million people every month with weight management services delivered by pharmacists who are highly trained professionals providing expert, accessible care. 'Despite this, the current NHS rollout remains extremely limited, with only a small number of eligible patients receiving support for obesity with weight loss treatments, and it is vital that this changes. 'With the right funding, pharmacies can scale up delivery quickly – ensuring more people get the help they need, when and where they need it.' News of the programme comes after Lilly published the results of a trial assessing a new daily pill for weight loss, which could offer a new and convenient alternative to jabs. People taking orforglipron lost an average of 12.3 kilograms in 72 weeks compared with those not taking the drug. The phased NHS roll-out of Lilly's weight loss jab Mounjaro to patients in England started in June for patients with a body mass index (BMI) over 40 and at least four co-morbidities. Over the next three years, about 240,000 patients are expected to be eligible for the treatment.
Medscape
3 hours ago
- Medscape
Growth Charts for Twins Improve Perinatal Risk Prediction
TOPLINE: New customised growth charts for twins (GROW-T), adjusted for maternal characteristics, more accurately predicted a small-for-gestational age (SGA)-associated risk for adverse perinatal outcomes than charts for singletons (GROW-S). METHODOLOGY: Researchers developed customised growth charts for twin pregnancies and compared them with singleton charts to evaluate their ability to identify SGA twins who are at an increased risk for adverse perinatal outcomes. They evaluated 8457 twin pregnancies (16,914 foetuses) from 127 NHS hospitals in the UK from January 2015 to February 2025, representing approximately two thirds of all NHS units. A mixed-effects linear regression analysis was performed to determine pregnancy-specific optimal weights at 37 weeks. Researchers compared GROW-T with GROW-S by calculating rates of the SGA-associated risk for stillbirth and adverse neonatal outcomes, such as the need for resuscitation, an Apgar score < 7 at 5 minutes, admission to the NICU, or neonatal death, using generalised estimating equations. TAKEAWAY: At 37 weeks, the average optimal weight of a twin was 389 g less than that of an equivalent singleton foetus. By pregnancy, 24.6% vs 64.9% of twins were classified as those with SGA (< 10th centile) according to GROW-T vs GROW-S; when analysed by individual foetuses, 13.8% vs 44.4% of twins were classified as those with SGA according to GROW-T vs GROW-S. Twins designated as SGA on the basis of GROW-T (odds ratio [OR], 7.2; 95% CI, 4.8-10.8) had a higher risk for stillbirth than those designated as SGA on the basis of GROW-S (OR, 2.8; 95% CI, 1.9-4.1). Twins classified as those with SGA on the basis of GROW-T had an increased need for resuscitation (OR, 1.3; 95% CI, 1.1-1.7), a lower Apgar score (< 7) at 5 minutes (OR, 1.8; 95% CI, 1.2-2.6), higher NICU admissions (OR, 1.3; 95% CI, 1.0-1.6), and an increased risk for neonatal death (OR, 5.4; 95% CI, 1.3-23.5). IN PRACTICE: "Our results add to the increasing evidence for adoption of twin specific charts," the authors wrote. "While adoption of twin specific charts is timely, implementation ought to be accompanied by evaluation in practice to monitor safety in different health service environments," they added. SOURCE: This study was led by Jason Gardosi, MD, Perinatal Institute, Birmingham, England, and was published online on August 01, 2025, in the American Journal of Obstetrics & Gynecology. LIMITATIONS: Ultrasound estimates of foetal weight in twins can be systematically overestimated, potentially affecting the accuracy of growth assessments. Chorionicity was not reported in the study cohort; however, the difference between growth curves of monochorionic and dichorionic twins was considered too small to be clinically relevant. DISCLOSURES: No funding information was provided for the study. All authors reported being employees of the Perinatal Institute, Birmingham, England. This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.
