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Pharmaceuticals

BioMarin drug becomes first U.S. approved therapy for dwarfism

Source: Reuters

Nov 19 (Reuters) – BioMarin Pharmaceutical Inc’s (BMRN.O) once-daily injection for children with the most common type of dwarfism received clearance from the U.S. health regulator on Friday, making it the first approved therapy for achondroplasia in the country.

The approval for Voxzogo, intended for children aged five and older, comes months after it became the first therapy to be greenlighted by regulators for the treatment of the genetic condition in Europe.

Many countries still do not have an approved therapy for Achondroplasia, a condition characterized by short stature which occurs in about one in every 25,000 newborns worldwide.

BioMarin expects the therapy’s annual net revenue per patient in the United States to be about $240,000 and expects about 3,000 children with achondroplasia in the country to be eligible for the treatment. Voxzogo is expected to be available in the United States by mid-to-late December at a list price of $899 per vial, the company said.

BioMarin’s late-stage Voxzogo study showed improvements in patients’ growth rate over a year and was well tolerated. The drug is also being studied in children below five years and has already been approved in Europe for those aged two and above.

The U.S. Food and Drug Administration had in April extended the review of the drug by three months to assess two-year data on the drug.

It has now cleared the drug under the accelerated approval pathway, which requires a post-marketing study assessing final adult height.

BioMarin said it intended to use the results from ongoing extension studies to receive final approval.

The drug is also under regulatory review in Japan, Brazil, and Australia and could receive approval in the countries next year, BioMarin said.

Reporting by Amruta Khandekar; Editing by Aditya Soni and Vinay Dwivedi

Our Standards: The Thomson Reuters Trust Principles.

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Business

How 5G and AI are shaping the future of eHealth

Global Director for AI/ML Solutions, Mona Nia Tecnotree

The digital transformation of the healthcare industry continues to gain momentum. This shift can be attributed to the rapid advancement of widely applied technologies such as 5G networks, cloud computing, artificial intelligence (AI), and big data.

Moreover, integrating 5G networks with cloud-based healthcare platforms and AI is driving the emergence of intelligent eHealth technology, projected to reach $208 billion by 2030, according to recent reports. Recent research by Grand View Research emphasises that the synergy between 5G and AI is pivotal in transforming healthcare by enabling faster data exchange, reducing latency, and improving the reliability of health solutions. This collaboration aims to revolutionise the healthcare sector by facilitating hyper-personalisation, optimised care, enhanced sales and services, and streamlined operations. Leading venture firms actively invest in healthcare start-ups using AI, fostering a rapidly growing ecosystem of innovative advancements.

As AI and 5G continue to make waves through all industries, healthcare needs to adapt to changes quickly. However, with operational, security, and data privacy concerns, healthcare organisations remain wary. As such, they must analyse their current and future needs to understand how AI and 5G technologies can help fulfil them and establish a comprehensive plan to guarantee its efficient and secure implementation in their practices.

Recent research by the International Data Corporation (IDC) emphasises that the synergy between 5G and AI could potentially reduce operational costs by up to 20% and improve patient outcomes by enabling more accurate diagnostics and personalised treatments.

5G Integration in eHealth

5G technology stands at the forefront of healthcare reform with its superior data speed and dramatically reduced latency. Tailored to concurrently accommodate multiple connected devices such as sensors, wearables and medical equipment, 5G is truly indispensable in healthcare, allowing IoT devices to seamlessly transmit accurate data for healthcare providers.

It empowers healthcare professionals to handle large, high-definition files like clinical visuals, videos, and real-time patient insights. 5G’s capability for network slicing—dedicating specific network segments for certain uses—simplifies the management of such files. In addition, it optimises the performance of each application, thereby removing the strain on medical staff.

However, the implementation of 5G technology shouldn’t be oversimplified. It’s essential to analyse the potential risks and challenges thoroughly. A principal component to consider is regulatory cybersecurity and data privacy. Given that 5G networks are susceptible to cyber attacks, it falls upon healthcare providers to protect data such as patient information.

Organisations should also consider the financial implications of implementing 5G technology, as it involves a considerable investment in infrastructure and equipment. Therefore, they must balance the potential gains against the costs to ensure the viability of the investment.

Recent discussions at Mobile World Congress 2024 highlighted the critical role of regulatory frameworks in ensuring the secure deployment of 5G in healthcare. Experts advocated for robust cybersecurity measures and collaborative efforts between technology providers and healthcare institutions to mitigate potential risks.

Marrying 5G and AI for Improved eHealth Solutions

Despite the challenges, integrating 5G and AI will pave the way for unprecedented growth within the internal medical ecosystem, enhancing healthcare quality and patient results. For example, deploying data to carry out descriptive-predictive-prescriptive analytics and transmitting the acquired insights using 5G can drastically improve the user experience while helping make informed decisions. Such an approach can assist healthcare organisations in identifying promising healthcare use cases like remote patient monitoring, surgical robotics, and telemedicine.

Moreover, AI-facilitated hyper-personalisation, driven by the profusion of data accessible through 5G networks, can evaluate patient histories, genetic profiles, and lifestyle elements alongside real-time vitals to prescribe tailored advice and treatments. AI can also automate scheduling appointments, streamline supply chain management, and enhance transactions such as claims and prior authorisations. AI-powered chatbots and virtual assistants can deliver real-life support, while patient and customer service applications can provide an enriched experience through increased data accessibility.

AI can also streamline healthcare services by predicting and managing disease outbreaks. Supported by 5G’s capacity for real-time operability, AI systems can instantly analyse patient data, oversee bed availability, and notify medical personnel of potential complications—promoting efficient, effective care delivery.

Finally, AI-empowered fraud detection algorithms operating on 5G networks can analyse copious amounts of data in real time to detect suspicious activities and alert responsible security teams. This can also be applied to security cameras that can detect anomalies in patients’ and visitors’ behaviour and notify appropriate staff members.

A study published in the Journal of Medical Internet Research (JMIR) in 2023 demonstrated that combining AI and 5G in telemedicine significantly improved patient satisfaction and reduced consultation times by 30%.

Shaping an AI Blueprint for 5G eHealth

Integrating AI and 5G technologies can revolutionise disease assessment and surveillance, facilitating more precise diagnostics and tailored treatments. In return, it will drastically improve the standard of care, curbing expenses and boosting efficiency.

Over the next few years, healthcare providers should focus on specific areas where 5G and AI can deliver the most impact. For example, developing telehealth platforms that excel in security, accessibility, and user-friendly interfaces will be paramount. This design aspect is set to thrive, particularly with 5G paving the way for high-definition video consultations, remote patient monitoring, and instant data sharing between patients and healthcare

providers.

The precision and availability of diagnostic applications powered by AI and tele diagnostic services will notably increase in tandem with the widespread adoption of 5G. The strategic emphasis should be on enriching its capabilities, ensuring compatibility with existing systems, and seamlessly integrating the tech into existing healthcare processes.

AI-guided care management systems will also play an integral role in eHealth. There is a need to structure these systems to constantly monitor patient progress, suggest highly personalised treatments, and coordinate care across multiple providers while prioritising patient privacy and data protection.

Finally, when it comes to home health monitoring, emphasis should be placed on creating IoT devices that can integrate seamlessly with AI-driven health platforms and securely transmit data; this will be a critical development within the field.

The synergy between 5G technology and AI will continue revolutionising the healthcare industry, offering more customised, efficient, and cost-friendly solutions. By developing a precise AI blueprint for critical eHealth applications and capitalising on the capabilities of 5G, the benefits will drastically outweigh the challenges.

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Lifestyle

Summer running: expert tips to safeguard your eyes from heat and sun

Running, as one of the most natural forms of recreation, comes with a long list of health benefits such as better cardiovascular health, improved knee and back health, better immunity, improved mood and overall energy, and better sleep.

Summer, despite being considered a nice time for a run due to the sunny weather, is the most hated season in the running community.

Running in the heat takes extra energy and it can take a toll on eye health. Prolonged exposure to UV rays can cause cataracts, macular degeneration, and pterygium (growth on the eye’s surface), while sweat and sun cream can lead to eye pain and blurred vision.

Nimmi Mistry, professional service optician at Vision Direct, shares insight on how to protect your eyes during summer runs.

Blinded by the lights: UV ray exposure can lead to serious eye damage

Running enthusiasts usually have their summer runs in the morning or evening, as these periods have lower temperatures. Despite the sun being lower then and runners looking down during their runs, indirect sunlight can cause serious eye damage as it reflects from surfaces.

Our eyes have natural protection, as corneas absorb UV-A, UV-B, and UV-C radiation, but they can absorb only half of it. The rest reaches the retina, which is responsible for sharp vision and the macula, and can cause damage such as:

Pterygium or Surfer’s Eye. This condition includes tissue growth in the eye that is directly connected to long-term exposure to UV light and can affect anyone that spends a lot of time outdoors without adequate protection. You can actually see this as a lumpy growth in your eye and although it’s not cancerous, it can cause eye inflammation, lead to dry eye, and cause other eye issues like itching, burning eye pain, or even lead to blurred and double vision. The only treatment for it is surgery.

Cataracts: With age, proteins and fibers in the lens begin to break down, causing changes in the eye tissues. Cataracts are basically little clouds in the eye lens that can cause vision problems and typically start appearing around age 40.  Studies have confirmed that UV rays can trigger the eye damage seen in cataracts, as they harm the proteins in the eyes in the same way. Prolonged exposure to the sun may cause your eyes to deteriorate even before you are 40.

Macular Degeneration: This is another age-related eye condition in which the macula, the part of the eye responsible for seeing sharp details, gets damaged. It’s common in people 65 years and older, and while the direct causes are not completely understood (generally bad habits like smoking, high cholesterol, and high blood pressure), studies have proven that exposure to UV rays increases the risk of developing macular degeneration.

Salt and acid from sweat leads to eye irritations 

Eyebrows are our natural barrier against getting sweat into eyes, but during summer runs they are not enough. The burning feeling of getting sweat mixed with sunscreen in your eyes isn’t just a temporary setback to your running pace.

Sweat that drips from your scalp to your eyes contains salt and acid, and can be potentially harmful. The salt in sweat can cause eye irritations, a burning feeling, blurred vision, or stinging eyes. If the sweat is mixed with sunscreen, the pain can be even sharper.

How to protect your eyes when running in the summer?

Whether you’re running on trails or on roads, you should definitely take care of your eyes, especially if you’re a long distance enthusiast.

  1. Wear sunglasses while running: This may seem logical but loads of runners still think running with glasses is uncomfortable. This may be true if they are wearing prescription glasses, but not if they wear specific lightweight sunglasses designed for outdoor activities. Wearing sunglasses is really imperative when it comes to eye protection as they block out 99 to 100% of UVA and UVB rays.
  2. Switch to contact lenses: If you’re wearing prescription glasses, seriously consider switching to contact lenses for sports or any summer activities. They will allow you to wear sunglasses, and with contacts, you also get additional UV protection and better peripheral vision. Daily contact lenses are the best choice for beginners, and they don’t have as much protein build-up, making them more comfortable to wear.
  3. Wear a running hat: The first choice for eye protection during summer is sunglasses, but if for any reason you can’t wear them, a running hat with additional UPF protection is a must. It will protect you from direct UV rays, but not from indirect UV rays, which again, can be really harmful. A running hat can be a nice addition to sunglasses, as it keeps sweat (combined with sunscreen) from dripping down and impairing your vision.
  4. Headbands: Headbands alone can’t protect your eyes from the sun, but it is a good idea to combine them with sunglasses to stop sweat and sunscreen getting into your eyes.

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Pharmaceuticals

Digital solutions offer key to better organisational efficiency and coordination in healthcare

Executive Chair of InnoScot Health, Graham Watson analyses the huge value which existing tech can realise for NHS Scotland

Healthcare innovation headlines are often made by the most impressive technologies – the likes of artificial intelligence (AI) and predictive medicine immediately spring to mind.

They capture the imagination, and rightly so given the exciting possibilities they offer and positive results which they are already producing, but of course they are not the entire solution. Instead, they represent effective pieces in the bigger puzzle that will eventually become the future of healthcare.

What also creates news headlines is spending that seeks to address the most pressing NHS concerns. Yet, such solutions do not always achieve the required result.

Akin to a leaking pipe, you can patch the hole in the short-term and the dripping immediately stops, but that does not mean the patch is necessarily an enduring solution. You might need to invest in an entirely new pipe.

In other words, by focusing on short term solutions, we may be left less able to focus spending on longer-term transformational change.

Grassroots thinking is also required then, and what is often less talked about is what is available in the here and now that can produce lasting results.

If the NHS is to do more with less and fundamentally work smarter, then we need to look at the tools that are already at our disposal and which do not require significant fresh resource – just more targeted approaches to how they are implemented and used.

That includes digital solutions for better leveraging of data to ensure that patients receive coordinated, seamless care; information shared quickly and securely between health professionals throughout the care journey; and digitally trained clinicians being increasingly unburdened of administrative processes allowing them to better focus on caring for their patients.

In essence, greater efficiency, systemic sustainability, less siloed systems, and giving back to clinicians that which they increasingly have less and less of – time.

If that digital shift in processes is managed effectively and then continuously monitored to identify potential improvements, it also likely translates to improved staff retention with clinicians able to make more confident decisions amid today’s challenging, often pressured work environment.

Secure and centralised cloud-based systems offer real time analytics, providing an at-a-glance dashboard of patient progress, including diagnoses, tests, and treatments, in tandem with the best possible accessibility across Scotland’s often nuanced healthcare system.

Most people are now familiar and comfortable with storing sensitive information securely in the cloud given just how much of our day to day lives are now kept there – and clinicians are no different, making NHS adoption a relatively straightforward culture change.

Indeed, there are great clinician-led examples already making waves, underlining the vast potential for existing solutions to be leveraged. Dr Matthew Freer, a consultant anaesthetist, is also CEO and co-founder of Infix Support – a cloud-based tech company focused on improving the efficiency of surgical operating theatres and tackling patient wait lists. His role in honing a more intuitive system for operating theatre utilisation is considered to be a game-changer.

Last year, InnoScot Health revealed the results of its independent survey of NHS Scotland workforce attitudes towards innovation, finding a marked enthusiasm to engage with new approaches and utilise technology to aid processes.

A total of 88% ranked big data and analytics – using gathered data to uncover hidden patterns and correlations for better decision-making and greater efficiency in healthcare delivery – as one of the most important areas for innovation in the future.

Also ranking 88%, digital apps – delivering information for patients, care providers and researchers, real-time patient monitoring, collecting community and clinical health data, and more – was a further prime focus for the workforce.

During this time of recovery and renewal, rapidly scalable digital innovations shaping the future of NHS Scotland must be integrated into its constantly evolving systemic architecture – exactly where they can have greatest impact – breaking down barriers, improving lives, and enhancing organisational efficiency.

When negativity can often predominate in NHS-related headlines, it is encouraging to note that we have a workforce responding positively and saying it is ready for a modern, agile, and sustainable health care system.

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