7^th Global Congress on Rare Diseases & Orphan Drug June 18-19, 2020 Dubai, UAE

Allergy is one of the most adverse immune reaction faced by worldwide   population which is next accompanied by inflammation. The set of conditions caused by Hypersensitivity reactions of the immune system in response to any foreign substance is called allergy/ allergic diseases. It may arise due to environmental or genetic factors. The causative agents are known as allergen and they may be food, drug, dust, pollen, animal dander &fur etc. varying for each individual. Most allergen immunotherapies  are the advanced therapeutics method to treat the allergies. Allergic and Inflammatory Reactions are caused by the immune system but if the reaction becomes adverse then the condition becomes chronic for an individual. The causative agents for such adverse reactions could be genetic, environmental, pathogenic and synthetic compounds. The localized body part which gets hot, swollen, red and painful after an injury or infection is an condition termed as inflammation and it might be due to allergy: Allergic inflammation or due to tumor. Inflammation can be   chronic or acute and it consists of different types based on the part of inflammation.

The majority of rare diseases affect children, most of whom have an underlying genetic cause for their condition. However, making a molecular diagnosis with current technologies and knowledge is often still a challenge. Pediatric genomics is rapidly evolving field but it is not well developed, that tackles this problem by incorporating next-generation sequencing technologies, especially whole-exome sequencing and whole-genome sequencing, into research and clinical workflows. This difficult multidisciplinary approach, combined with the ascending availability of population genetic data variation, has already resulted in an increased discovery rate of causative genes and in improved diagnosis of rare pediatric disease. Substantially, for affected individual family members, a good and thorough understanding of the genetic basis of rare disease translates to more perfect prognosis, management, surveillance and genetic advice; stimulates research into new therapies; and enables provision of better support.

Many of the terms are normally used in describing the infectious diseases.  Disease refers to the presence of pathology and an infectious disease is an infection caused by a microorganism. Virulence is a term referring to the power of an organism to produce disease in a particular host. Immunity refers to the degree of resistance of the host for a specific microbe.  Many infectious maladies, such as measles and chickenpox, can be anticipated by vaccines. Infectious diseases can cause many distinctive symptoms. Some are mild that you may not even noticed the any side effects, while others can be the life-threatening. There are a medicinal products for a few infectious diseases, but for others, such as a few viruses, you can only treat your symptoms. You can take steps to prevent many infectious maladies. There are numerous  ways that you can get an infectious disease for example – through  animal or insects  bites, contaminated food, and water. Pregnant womens can too pass some germs along to their babies; the infectious diseases are usually characterized by the dominant organ system involved.

A clinical trials are the experimental designed to determine various scientific and consequence aspects of proposed treatment. It is important to remember that there is no guarantee a proposed treatment will be effective. Families should be prepared to balance the hope of participating in a clinical trial with the understanding that the therapy may not be optimal and could result in a serious negative outcome. Since some rare diseases can progress quickly, families often are willing to participate in risky, unknown treatments. Before agreeing to participate, everyone involved should fully understand these risks by carefully reading the consent documents and asking questions of the trial investigators. Most clinical trials related to treatments of rare diseases goes through 3 major phases.

Neurological disorders are the diseases associated with the spine, brain and the nerves that connect them. It affects the body nervous system. Aicardi-Goutieres, Reflex Sympathetic Dystrophy, Acarida syndrome are few rare specified neurological disorders to specify. The central nervous system affected by Rare Neurological Disorders usually develops in infancy and is inherited in an autosomal recessive manner. There are various kinds of symptoms which are categorized in three main stages that provide a general basis for diagnosing individuals with LS. The symptoms of the stage I are characterised as stunted growth, vomiting, and diarrhoea.  Symptoms exhibited in stage II are example such as optic atrophy, uncoordinated movement, dystonia, hypotonia, and peripheral neuropathy. The symptoms of stage III are dysarthria, acute muscle and dysphagia, atrophy is common, with death being the outcome. It is necessary to study the physiological defects of brain and muscle structures in LS for proper diagnosis and treatment.

 Neglected tropical diseases are a bunch of bacterial and parasitic infections that affect more than including 1 billion population and cost developing economies billions of dollars each year, 836 million children. They are regularly as possible clustered together topographically and people are habitually afflicted with more than one contamination or parasite. Successful controls can be accomplished when chosen open wellbeing approaches are combined and passed on locally. Trade are guided by the local epidemiology and the accessibility of fitting measures to recognize, expect and control diseases. Various dismissed tropical diseases can be expected, disposed of or indeed demolished with made strides get to existing secure and cost-effective gadgets. Control depends on straightforward mediations that can be carried out by non-specialists too.

Gene Therapy mainly involves the introduction or alteration of genetic material within a cell or organism with an intention of curing the disease. Both gene therapy and cell therapy are overlapping fields of biomedical research with the goals of repairing the direct cause of Genetic diseases in DNA or cellular population respectively, the discovery of recombinant DNA technology in the 1970s provided tools to develop gene therapy efficiently. Scientists use these techniques to readily manipulate viral genomes, isolate genes and identify mutations involved in human diseases, characterize and regulate gene expressions, and engineer various non- viral and viral vectors. Various long-term treatments for anaemia, haemophilia, cystic fibrosis, muscular dystrophy, Gauscher’s disease, lysosomal storage diseases, cardiovascular diseases, diabetes and diseases of bones and joints are resolved through successful gene therapy.

Hematology of rare diseases fall into various subcategories, including anaemia-type red blood cell conditions (e.g. sickle cell beta thalassemia, Diamond-Blackfan anemia), white blood cell dysfunctions related with different malignancies ,immuno- platelet-based abnormalities that influence platelet and plasma cell disorder. Some rare blood disorders especially those that are gene-based are simple to distinguish with a basic test, whereas others require significant investigator work to effectively analyze. Due to the expansive number of blood conditions   exceptional and common, haematologists tend to be gifted agents since they got to be pros in different conclusion.

Obesity can be preventable through a combination of social changes and personal choices. Diet plan and exercising are the main treatments. Diet quality can be improved by doing less consumption of energy-dense foods, such as those high in fat or sugars, and by increasing the intake of Fibrous food and rich in all type of protein.  Various ways can be adopted to reduce appetite or decrease fat absorption by Medications, along with a suitable diet. If these ways such as diet, exercise, and medication are not effective, a gastric balloon or surgery may be performed to reduce stomach volume or length of the intestines, leading to feeling full earlier or reduce the ability to absorb nutrients from food.

Metabolic disorders can be present at birth, and many can be identified by routine screening of the body. If a metabolic disorder is not identified in the early stage, then it may be diagnosed later in life, when symptoms appear. The genetic metabolic disorders can be diagnosed by specific blood and DNA test. The gut microbiota, which is a population of microorganisms that live in the human digestive track, also has an important part in metabolism and generally has a positive function for its host where they live. In terms of pathophysiological/mechanism interactions, the abnormal gut microbiome can play a role in metabolic disorder related to obesity.