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May 12.2025
3 Minutes Read

New Hope for Cystic Fibrosis and Amyloidosis with European Drug Approval

Alyftrek package for Orphan Drugs for Cystic Fibrosis and Amyloidosis.



Positive Developments for Cystic Fibrosis and Amyloidosis Patients

In a significant advancement for patients suffering from cystic fibrosis (CF) and hereditary transthyretin (TTR)-mediated amyloidosis, the European Medicines Agency (EMA) recently recommended approval of two new orphan drugs. These treatments are crucial as they offer hope for improved quality of life and disease management in these underserved populations.

Revolutionary Treatments on the Horizon

The EMA's nod to Alyftrek, a combination therapy by Vertex Pharmaceuticals, aims to change the lives of patients aged six and older with at least one F508del mutation in the CFTR gene. This combination medicine, consisting of deutivacaftor, tezacaftor, and vanzacaftor, works synergistically to restore functionality to the CFTR protein, which plays a crucial role in maintaining healthy lung function.

Clinical trials demonstrated that Alyftrek significantly improved lung performance—offering a lifeline for many who were previously reliant on inhalers and other therapies for symptom relief. The dual approach of targeting different sites on the CFTR protein enhances its efficacy compared to existing treatments like Kaftrio, making this innovation noteworthy in the fight against cystic fibrosis.

A Breakthrough for Amyloidosis

Meanwhile, Attrogy has emerged as a beacon of hope for those dealing with hereditary TTR-mediated amyloidosis. This condition, characterized by gradual nerve damage and organ dysfunction, has long been a challenge for patients. The drug, which stabilizes the TTR tetramer, aims to counteract disease progression effectively.

In clinical trials, Attrogy delayed degeneration as assessed by the Neuropathy Impairment Score plus 7 nerve tests (NIS + 7), showcasing its potential to transform standard care for patients. By addressing both the symptoms and underlying causes of the disease, Attrogy represents a significant stride forward in patient-centric care.

Understanding Orphan Drug Designation

Both Alyftrek and Attrogy received orphan drug designation, which is a recognition intended to stimulate research and development in areas with limited therapeutic options. The implications of approval extend beyond just new medications; they signify a strong commitment to prioritize and uplift patients battling rare diseases.

Next Steps and Future Implications

As the EMA prepares to finalize marketing authorizations, healthcare professionals and patients are urged to stay informed about the potential side effects and monitoring requirements that accompany these medications. With ongoing studies and clinical data collection, these drugs will not only enrich therapeutic options but could also pave the way for future medical innovations in rare disease treatments.

Community Impact and Patient Empowerment

For suburban professionals between 25-50 years, particularly those with loved ones diagnosed with CF or amyloidosis, the news of these drugs represents not just medical progress, but also a step toward empowerment. Increased awareness and advocacy can lead to better understanding and accessibility of treatment options.

The broader health community is encouraged to advocate for improvements in medical research funding, which can drive the development of more orphan drugs. Staying informed about the latest medical trends is essential, as it not only advocates for personal health but also supports a larger conversation around wellness for all.

In closing, the approval of Alyftrek and Attrogy heralds a new era of hope for patients facing challenges from cystic fibrosis and hereditary amyloidosis. As these treatments gain approval, and ultimately begin to reach patients, the optimism surrounding health and wellness continues to grow.

For healthcare professionals and patients alike, keeping abreast of these advancements is essential. By understanding how these drugs will impact treatment protocols, individuals can make informed decisions that enhance their or their family member's well-being.



International Health News

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06.08.2025

Why the UK Faces a Severe Creon Shortage and Its Impact on Patients

Urgent Crisis: The Creon Shortage in the UKIn an alarming development, nearly all pharmacies in the UK are facing a severe shortage of Creon, a critical pancreatic enzyme replacement therapy (PERT). The National Pharmacy Association (NPA) has raised the alarm, revealing that a staggering 96% of pharmacies reported difficulties in obtaining this essential medication. Moreover, 89% of pharmacies highlighted challenges in providing alternative PERT solutions to their patients, sparking concerns about the ramifications for those who rely on this life-sustaining treatment.The Impact on Patients and Their Well-BeingThis ongoing crisis has forced patients into dire circumstances. Many individuals are resorting to rationing their medication supplies or are skipping meals entirely in a desperate bid to manage their health. One pharmacist described the situation as the “worst stock shortage” they have ever experienced, while patients share stories of traveling long distances or contacting numerous pharmacies to find limited supplies.Creon itself is not just another medication; it’s integral for individuals suffering from pancreatic exocrine insufficiency due to conditions like cystic fibrosis, pancreatic cancer, or chronic pancreatitis. Its formula, which includes lipase, protease, and amylase, is designed to facilitate digestion. The lack of access to this drug can lead to an array of serious symptoms, including abdominal pain, weight loss, and debilitating digestive issues, that can severely diminish quality of life.Navigating the Shortage: Government Response and Patient SafetyOlivier Picard, NPA chair, voiced the growing disappointment, stating that it is unacceptable for patients to ration their medication in the 21st century. Responding to the situation, the UK government has extended the Serious Shortage Protocol (SSP) for Creon until November 2025. Under this protocol, pharmacists are permitted to exercise professional judgment when substituting prescriptions, allowing for some flexibility in this critical time. However, a survey indicated that 81% of pharmacies felt these measures were insufficient, often requiring them to send patients back to prescribers when they sought alternative PERT options.The Challenge of Finding AlternativesAdding to the complexity of the situation, alternative PERT products such as Nutrizym and Pancrex are also facing shortages as manufacturers struggle to keep up with the skyrocketing demand. While GPs and pharmacies have the option to import unlicensed Creon or other PERT products from overseas, the global nature of these shortages makes this a delicate and often unreliable solution.A Call for Regulator InterventionThe crisis highlights the need for immediate action from health regulators and pharmaceutical companies to address the underlying issues causing these shortages. By ensuring a steady supply of Creon and its alternatives, they can help safeguard the well-being of thousands of patients across the country.Future Considerations and Broader ImplicationsThe Creon shortage is not just a logistical issue; it’s a human one that illustrates the fragile state of our healthcare supply chains. This crisis could pave the way for significant changes in how medications are sourced, monitored, and distributed, emphasizing the importance of building more resilient systems that can better serve vulnerable populations in times of need.As the health and wellness community monitors this unfolding situation, it’s crucial to advocate for patient rights and access to necessary medications. It’s a stark reminder of the essential role pharmacies and healthcare professionals play in our well-being.In closing, we must foster community support and dialogue to help identify viable solutions to ensure that no one is left without access to life-saving treatments like Creon. Share your experiences, spread the word, and call for action towards a more sustainable and equitable healthcare landscape.

06.05.2025

Unlocking Hope: CAR T-Cell Therapy Dramatically Improves PFS in Advanced Gastric Cancer

CAR T-Cell Therapy: A Breakthrough for Advanced Gastric CancerA new phase 2 clinical trial has revealed that satricabtagene autoleucel (satri-cel), a type of CAR T-cell therapy, significantly boosts progression-free survival (PFS) in patients suffering from advanced gastric and gastroesophageal junction cancers. These patients, who tested positive for the Claudin-18 isoform 2 (CLDN18.2) protein indicating aggressive malignancy, previously had limited treatment options, often facing grim prognoses.The Study: Methodology and Key FindingsThis randomized controlled trial, conducted across various centers in China, involved 156 participants with pathologically confirmed CLDN18.2-positive advanced diseases that had resisted multiple standard treatments. Participants received either the cutting-edge satri-cel therapy or treatment of physician's choice (TPC), which consisted of several conventional chemotherapy agents.The results were promising: patients receiving satri-cel had a median PFS of 3.25 months compared to just 1.77 months for those in the TPC group. Additionally, the objective response rate was markedly higher in the satri-cel group, achieving 22% versus only 4% in the control group. The data illustrated an impressive 63% reduction in the risk for disease progression or death.Understanding CAR T-Cell TherapyCAR T-cell therapy is a novel treatment method where a patient's immune cells are engineered to better recognize and attack cancer cells. For patients with advanced gastric cancers, where traditional therapies often fail, this innovative approach offers new hope. Satri-cel specifically targets the CLDN18.2 protein, found to be overexpressed in gastric malignancies. The treatment showcases a shift towards personalized medicine, where therapies are tailored based on the unique characteristics of a patient’s cancer.Life After Diagnosis: The Human ImpactThe emotional strain of being diagnosed with advanced cancer can be overwhelming, often leaving patients and families grappling with fears of mortality and questions about quality of life. The advent of effective treatments like satri-cel can transform this narrative, offering not just longer survival but also improved quality of life. Many patients share anecdotes of renewed hope and vitality after receiving innovative treatments that finally started working where others have failed.Looking Ahead: What’s Next for CAR T-Cell Therapy?While the results of the clinical trial are promising, researchers caution that further validation is essential. Future studies will focus on long-term outcomes to determine the efficacy and safety of satri-cel beyond initial treatments. Additionally, this research lays the groundwork for potential adaptations of CAR T-cell therapies to target other cancers, broadening the scope of this transformative technology.The Future of Treatment OptionsIn light of these advancements, it’s crucial for patients to stay informed about emerging therapies and advocate for themselves within the healthcare system. The innovative nature of CAR T-cell therapy may soon expand, leading to customized treatments that work better for various individual patients based on their unique cancer profiles.Final Thoughts: Embracing New Innovations in Cancer CareFor those navigating the often difficult landscape of cancer treatment, breakthroughs like satri-cel signify a beacon of hope. The ongoing advancements in medical research not only look to improve survival rates but also enhance the overall well-being of patients facing advanced cancers. Keeping abreast of these developments can empower individuals to make informed choices and remain hopeful about the future of their health.

06.03.2025

Why UK Hospitals Must Implement Frailty Screening for Surgery Patients

Why Frailty Screening in Surgery Is Crucial for Older PatientsIn an alarming revelation, a large-scale study has shown that nearly three-quarters of UK hospitals are missing a critical step in pre-surgical care: screening older patients for frailty. Conducted by the Royal College of Anaesthetists (RCoA) in conjunction with the University of Nottingham, this research reveals that 71% of hospitals do not routinely assess patients over 60 for frailty, despite the significant risks associated with this condition.Frailty, which affects about 1 in 5 surgical patients aged over 60, drastically increases the likelihood of postoperative complications, extended hospital stays, and even mortality. The findings highlight a troubling trend in healthcare that could potentially hinder recovery for an increasingly aging surgical population. Acknowledging the seriousness of the situation is the first step towards improving patient care.The Current Landscape: A Call for ActionThe Sprint National Anaesthesia Project (SNAP3) data presents stark statistics: elderly patients suffering from frailty are three times more likely to suffer complications and four times more likely to experience postoperative delirium. To confront these alarming outcomes, researchers propose that frailty assessments should become a standard practice before surgery. This would not only enhance patient safety but fundamentally shift the approach to surgical care in the UK.Dr. Claire Shannon, the president of the RCoA, voices the urgency for systematic frailty screening, stating that such measures could significantly streamline patient recovery and decrease unnecessary hospital stays. “As older individuals constitute an increasingly substantial part of the surgical demographic, effective frailty screening becomes imperative,” she asserts.Understanding Frailty in Surgical ContextsFrailty is not merely an age-related condition; it's a multifaceted concern that can stem from various health issues, influencing how patients respond to surgical procedures. According to the research, frail patients spend an average of three additional days in the hospital post-surgery, while severely frail individuals can face an extension of up to six days. This underscores an urgent need for increased clinician awareness and a more proactive approach in identifying vulnerable patients ahead of surgical interventions.With evidence supporting the dire consequences of overlooking frailty, clinicians must be equipped with tools such as the Clinical Frailty Scale, enabling them to identify at-risk patients early on. This tool offers a straightforward assessment procedure that can lead to better patient preparation and engagement in post-operative care.Collaborative Care: Key to Successful OutcomesCollaboration among medical professionals is crucial in managing frail patients. Iain Moppett, chief investigator of SNAP3 and an esteemed professor, emphasizes that recognizing frailty must foster collaborative dialogues among surgeons, anaesthetists, and geriatricians. The goal is to enhance overall patient wellness pre-surgery and facilitate informed decision-making tailored to individual circumstances.This approach advocates for forming cohesive teams committed to ensuring that frail patients receive the optimal support they need, allowing them to enter procedures in the best possible condition. The ripple effect of such improvements extends beyond individual patients to the healthcare system as a whole, optimizing resources and reducing pressure on hospital staff.Transitioning Toward Better Patient OutcomesThe collective impact of these insights calls for a cultural shift in how surgical care is approached, particularly for older patients. By emphasizing frailty screening and promoting interdisciplinary collaboration, the healthcare system can mitigate risks and enhance outcomes for susceptible populations. As aware and informed as we must be, this proactive approach can pave the way for improved standards of care across the board.Enhancing Patient Independence and Well-BeingBeyond the immediate surgical context, understanding frailty and its implications is key to enhancing overall patient independence and well-being. With an expanding demographic of older adults, the healthcare strategy must evolve to meet their unique needs.By instituting routine frailty assessments, hospitals can not only ensure immediate safety but also contribute to long-term well-being, allowing individuals to maintain greater autonomy in their later years. As awareness grows and practices evolve, patients can expect more informed, compassionate, and comprehensive care.Conclusion: The Path ForwardFor older patients, the necessity of frailty screening cannot be overstated. As we glean insights from the SNAP3 study, it becomes clearer that implementing universal frailty assessments is essential for optimally caring for surgical patients. As trends in health care continue to evolve, prioritizing these assessments will not only enhance recovery results but also foster a culture of wellness and vigilance within the medical community.Now is the time to advocate for change within the surgical framework for older patients. Be proactive in discussing frailty assessments with healthcare providers, utilizing tools and techniques that can significantly improve outcomes. When it comes to health and wellness, taking that first step could be transformative.

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