Pharmaceutical agents which treat rare medical conditions like orphan or rare diseases are called “orphan drugs”. The name ‘orphan’ itself indicates that the pharmaceutical industries are showing less interest in the development and marketing of drugs intended only for a small number of patients. Difficulty in the diagnosis and therapeutic management of rare disease is the major challenge in the development of orphan drug.
In recent years, progress has been made in the development of orphan drugs by pharma industries due to enactment of different regulations, administration authorities, tax benefits, marketing rights and public awareness by different countries. This review provides an overview of incentives, marketing rights and administrative authorities of different countries like U.S, European Union, South Korea, Japan, Australia and Taiwan.
Artificial Intelligence has exploded as a research subject in the 21st century as the hardware requirements of theoretical Artificial intelligence in the 19th and 20th century have translated into reality. This has led to rapid progress with the realization of multiple kinds of neural networks, as well as improvements along classical Machine Learning models like Decision Trees, Support Vector Machines etc. Most recently, the focus in Machine Learning has now shifted towards generative networks with Variational Autoencoders (VAEs) and Generative Adversarial Networks (GANs) leading modern research topics.
With advent of the Internet of Medical Things (IoMT) and modern time leaps in the domain of Artificial Intelligence with the explosion of Machine and Deep Learning, Medical AI has grasped the central stage in modern research. Applications of Medical AI vary from detections of various cancerous tumors to prediction of arrhythmia attacks. The augmentation of such AI embedded techniques into the medical profession has streamlined analysis as well as aided professionals in reaching a more efficient and accurate diagnosis. This has also translated in the field of dentistry where strong deep learning architectures such as Convolutional neural networks in the form of Resnet, Inception, GoogleNet etc have been used to process raw images and detect a range of dental diseases.
This paper aims to provide an overview of progress made in the field of Machine Learning particularly focusing on its medical and dental applications. Different facets of Machine Learning are discussed with respect to their strengths, shortcomings, and the way artificial intelligence has been used to tackle problems in the medical field. Finally, a descriptive overview about state-of-the-art machine learning reliant applications that are being used in different dental subfields is discussed along with current challenges the industry faces today.
Diabetic foot ulcers (DFU) are chronic wounds, which do not respond to traditional wound treatments. In this work, wound dressings of glibenclamide (GB) incorporated into a novel mixed matrix were fabricated in the aim of accelerating the healing process of diabetic wounds. GB was loaded into different weight ratios of Soluplus® (SP) and polyvinylpyrrolidone (PVP). The developed dressings were characterized İn vitro and in vivo, for their ability to promote diabetic wound healing. The particle size was
between (1.4-2) µm. The morphology abided by the SP/PVP ratio in the formulated microparticles. Cup/bowl shape, semispherical with corrugated surface, apple shape with smooth surface, concave/star shape, and Irregular corrugated morphology were denoted for GB-SP/PVP1-0, GB-SP/PVP1-1, GB-SP/PVP0-1, GB-SP/PVP1-2, and GB-SP/PVP2-1 formulations, respectively. Glibenclamide was in amorphous form and hydrogen-bonded with the matrix polymers. The GB-SP/PVP0-1 wound dressings showed a burst drug release in about 1 hour due to the hydrophilic nature of PVP. The other GB-SP/PVP formulated polymeric micelles were of sustained release, where GB-SP/PVP2-1 extended the drug release for 48 hours. The MTT assay showed that all GB-SP/PVP dressings have good cytocompatibility, and in consequence, they can be used in further investigations on biomedical applications. In vivo tests on a rat model of a full-thickness wound showed rapid closure, indicating the success of the wound dressings in decreasing inflammation and promoting wound healing without scar formation. Therefore, topical administration of GB-SP/PVP1-0 and GB-SP/PVP2-1 wound dressings has a high potential for the treatment of diabetic wounds in inflammatory and proliferative phases of healing with high bioavailability and fewer systemic adverse effects.
Background: The wide spread of COVID-19 disease and mortality associated with it as never seen before urged the medical research field to formulate and investigate the efficacy of various new drugs alongside with the existing drugs.
Objective: To evaluate the potential benefits of Tocilizumab (TCZ) in addition to the Standard of Care (SoC) in reducing mortality in moderate & severely ill COVID 19.
Methodology: Study population is sorted by 1:1 matching depending on the High-Resolution Computed Tomography (HRCT) Chest CT Severity Score. Outcome is measured as deaths, discharge with or without Long Term Oxygen Therapy (LTOT) along with clinical improvement of the patient measured using 9 points ordinary scale score of WHO and length of stay in Intensive Care Unit (ICU) post administration & levels of inflammatory markers (CRP, IL 6) on day of administration- baseline (D0), 1- & 3-days post administration (D+1 & D+3). The group received TCZ+SoC is considered as Test Group and the group received only SoC as Control Group.
Results: Deaths and discharge with LTOT were slightly higher in test group with OR 1.68 & 1.63 respectively. Clinical improvement in terms of 9-point ordinary scale score & difference in the levels of CRP & IL 6 was insignificant in both the groups. No difference in post administration ICU stay is observed between the groups.
Conclusions: Our study concludes no marked benefits with the addition of TCZ to the SoC in treating moderate and severe COVID 19 patients.
Background: Gene therapy has proved to be a boon for neuromuscular diseases. A concept introduced in the early 1960’s, has been put into clinical practice in the past 5-10 years. The process by which a healthy gene replaces a defective gene in a human body through vectors is truly pathbreaking. Patients with inherited degenerative neuromuscular disorders such as spinal muscular atrophy, and muscular dystrophies undergo progressive deterioration and eventually death. Gene therapy and other adjunctive clinical approaches have provided such patients with a second chance to truly live their life. This review is centered on the different gene therapies currently developed for spinal muscular atrophy and Duchenne muscular dystrophy and also sheds light on the emerging therapies.
Methods: Literature search was done in Medline, Embase, and Google Scholar. The relevant and important articles were included for developing the narrative review.
ResultsandDiscussion: The nusinersen, risdiplam, zolgensma, eteplirsen, golodirsen, viltolarsen, casimersen and ataluren are developed as gene therapies for Spinal muscular atrophy and Duchenne muscular dystrophy. The therapies approved under orphan drug designation have shown muscle strength improvement in clinical trials. However, long-term data on safety and efficacy is to be determined. The cost of the therapies acts as an impediment in most cases.
Conclusion: The development of gene therapies for inherited genetic disorders would be a prelude for several other genetic diseases. The studies in this arena would provide a prototype for translational research from bench to bedside. Further efforts are mandated towards long term safety, efficacy, and affordability of therapies.