Search
4 articles for “orphan disease”
-
Horizon Scanning and Early Assessment of Health Technologies for the Treatment of Orphan Diseases
Abstract: The theoretical foundations and regulatory framework of the processes of the formation of an effective health technology assessment (HTA) system at the early stages of the life cycle of medicines are analyzed in the article. Particular attention is paid to horizon scanning (HS) and early assessment of expensive innovative drugs utilized for treating rare diseases. In order to inform policymakers, purchasers, and providers (to prioritize MT research, financial, and operational …
Published in International Journal of Brain Sciences · Vol. 1, Issue 1, 2024 · pp. 32–39 Read article
-
Cancer Chemotherapy-Induced Cachexia: Bridging Mechanisms, Management, and Future Therapies
Abstract: Involuntary weight loss, skeletal muscle atrophy, adipose tissue depletion, anorexia, and systemic inflammation are all symptoms of chemotherapy-induced cachexia, a complex illness. Although it is common, it remains poorly understood and is often referred to as an "orphan disease." This study covers the epidemiology, pathophysiology, clinical symptoms, diagnosis, treatment, and emerging treatment options for chemotherapy-induced cachexia. To comprehend the causes, clinical results, and therapeutic approaches, a literature-based synthesis of recent …
Published in Research and Reviews: A Journal of Pharmacology · Vol. 16, Issue 1, 2026 · pp. 17–26 Read article
-
Transforming Rare Disease Diagnosis with AI
Abstract: Artificial intelligence is changing healthcare fast. It is making diagnoses accurate, helping doctors get better results, and streamlining how care works. This paper looks at how AI shows up in healthcare right now – where it is already making a difference, what is working, and what is still tricky. The focus is on machine learning, natural language processing, and computer vision. Particular attention is given to using AI in diagnosing …
Published in Research and Reviews : Journal of Computational Biology · Vol. 15, Issue 1, 2026 Read article
-
Bedaquiline: An In-Depth Exploration into Its Antitubercular Significance
Abstract: Tuberculosis is still a leading infectious disease and a major threat to public health. Although there has been a slow decrease in the incidence over years, its “hard to kill nature” is responsible of about 2 million deaths in HIV infected individuals annually. Bedaquiline, a Diaryl-Quinoline drug was fast-track approved as an orphan drug to combat drug resistance. It shows potent activity against Mycobacterium tuberculosis by interfering with the production …
Published in Research & Reviews: A Journal of Drug Design & Discovery · Vol. 12, Issue 2, 2025 · pp. 06–14 Read article