quote:"For most families, it’s a shock. Cystic fibrosis still has a strong association with being a life-threatening disease, despite the fact that we now have good treatments, and many patients can live healthy lives. The diagnosis puts a huge psychological strain on the family, especially when dealing with very young children.",
aimofcontact:"To gain a deeper insight into the path to diagnosis, we invited pediatrician Dr. Cristian-Gabriel Olariu from the University Department of Pediatrics and Adolescent Medicine to share his experiences with cystic fibrosis (CF) patients with us. We interviewed him because of his expertise in the effects of diagnosis on the patient and the family members, but also on daily life. Additionally, we want to close the gap and create a bridge between academic research and clinical applications. Therefore, Dr. Olariu gave insights about the clinical perspectives on CF patients.",
insights:[<p>We invited Max, our CF patient contact, to join Dr. Olariu in discussing the intersection of academic research, clinical application, and patient needs. Through our connection with CF Vests Worldwide (link zu deren Website? https://www.cfvww.org), an organization dedicated to providing life-saving therapy vests to cystic fibrosis patients globally, we gained insights into the challenges faced by CF patients, particularly in regions like Thailand, where access to advanced treatments and medical devices is limited. The conversation highlighted the critical role of early diagnosis and intervention, as well as the quality-of-life challenges many patients endure due to conventional treatments that may not be effective for everyone. Innovative approaches, such as our SORT LNP (lipid nanoparticle) delivery system, present promising alternatives for CF therapy. This system, which allows for RNA encapsulation and administration via dry spray inhalation, could revolutionize treatment by targeting lung cells more effectively, particularly in resource-limited settings. Dr. Olariu underscored the need for psychological support and coordinated care for CF patients, emphasizing that novel therapies like LNP-based gene treatments have the potential to improve treatment efficacy and accessibility, ultimately reducing the lifelong burden of care for patients and their families. </p>
insights:[<p>We invited Max, our CF patient contact, to join Dr. Olariu in discussing the intersection of academic research, clinical application, and patient needs. Through our connection with CF Vests Worldwide (link zu deren Website? https://www.cfvww.org), an organization dedicated to providing life-saving therapy vests to cystic fibrosis patients globally, we gained insights into the challenges faced by CF patients, particularly in regions like Thailand, where access to advanced treatments and medical devices is limited. The conversation highlighted the critical role of early diagnosis and intervention, as well as the quality-of-life challenges many patients endure due to conventional treatments that may not be effective for everyone. Innovative approaches, such as our SORT LNP (lipid nanoparticle) delivery system, present promising alternatives for CF therapy. This system, which allows for RNA encapsulation and administration via dry spray inhalation, could revolutionize treatment by targeting lung cells more effectively, particularly in resource-limited settings. Dr. Olariu underscored the need for psychological support and coordinated care for CF patients, emphasizing that novel therapies like LNP-based gene treatments have the potential to improve treatment efficacy and accessibility, ultimately reducing the lifelong burden of care for patients and their families. </p>,
<ol>
<li>Diagnosis:</li>
<li>Detection through newborn screening.</li>
<li>Further tests (including sweat tests) are conducted if results are abnormal.</li>
</ol>
</ol>,
<ol>
<li>Early Treatment:</li>
<li>Begins with inhalations, physiotherapy, and medications.</li>
<li>Aim: Prevention of severe complications and organ protection.</li>
</ol>
</ol>,
<ol>
<li>Challenges:</li>
<li>Some patients do not respond well to conventional treatments.</li>
<li>Significantly impacts quality of life.</li>
</ol>
</ol>,
<ol>
<li>Family Burden:</li>
<li>Medical challenges create a significant burden.</li>
<li>Psychological stress due to lifelong treatment.</li>
</ol>
</ol>,
<ol>
<li>Importance of Support: </li>
<li>Psychological support is crucial.</li>
<li>A well-functioning treatment team is essential.</li>
</ol>
</ol>,
<p>We have jointly weighed up the extent to which an early diagnosis is always an advantage, as some parents perceive an early diagnosis as an additional burden and would prefer to experience the first years of their child's life without constant medical intervention. Especially when there are cases in which patients only show a clear clinical picture at an advanced age. The psychological burden also lies with the children, who often experience medical trauma because they are involved in such intensive medical care from birth. Additionally, the treatment of cystic fibrosis is very expensive, and the costs are covered by health insurance companies to varying degrees. In some countries, such as the USA, Ukraine or Developing countries, many families cannot afford the necessary treatments. Dr Olariu drew our attention to another problem in the treatment of cystic fibrosis. Infections, especially with bacteria such as Pseudomonas spcc., are difficult to treat and often lead to long hospital stays. Max, our patients’ representative, who knows Dr. Olariu through his treatment, talked about his infections with Pseudomonas spcc., illustrating the reality of an invisible danger that determines a patient's everyday life. Strict hygiene measures are required to prevent infections, such as wearing face masks in hospital and careful handling of potential sources of infection. The clinics where cystic fibrosis patients are treated work closely with a multidisciplinary team of doctors, psychologists, physiotherapists and nutritionists to ensure that patients receive holistic care. At the same time, research is constantly being carried out and new therapeutic approaches developed, such as the use of nanoparticles to improve drug delivery. Former patients are also involved in research and provide valuable insights and advances. </p>
<ol>
,<ol>
<li>Pros of Early Diagnosis and Treatment</li>
<ol>
<li>Timely Intervention: Prevents severe organ damage and improves long-term outcomes.</li>
