scientific tracks

Track 1: Different types of Rare Diseases

A rare disease is defined as a condition that affects less than 200,000 people. This clarification was framed by Congress in the Orphan Drug Act of 1983. There may be approximately 7,000 rare diseases. When a person is diagnosed only a few categories of rare diseases are tracked. These include certain infectious diseases, cancers and birth defects. Because most rare diseases are not trailed, it is hard to determine the correct number of rare diseases. Most of the rare diseases are genetic, and thus are present throughout the person's intact life, even if symptoms do not immediately appear.

Track 2: Rare Infectious Diseases and Immune Deficiencies

About one-third of human deaths are attributable to infections across the world. Additionally, the so-called non-infectious causes of death often have a mysterious infectious etiology. Many rare diseases/orphan diseases caused by infectious agents rather than genetic or environmental factors.

Track 3: Rare Genetic Diseases/Disorders

A genetic disorder is a health problem caused by one or more abnormalities in the genome. It can be caused by a mutation in a single gene (monogenic) or multiple genes (polygenic) or by a chromosomal abnormality. Around 300-350 million people on earth are living with rare disorders - this is a disorder or condition with fewer than 200,000 people diagnosed. About 80% of these rare disorders are genetic in origin, and 95 percent of them do not have even one treatment approved by the FDA.

Track 4: Mystery Diagnosis of Rare Diseases

Diagnostic error in medicine is prevalent. For example, a study from an intensive care unit demonstrated nearly 20% discordance between the clinically-defined cause of death and findings at post-mortem examination. Not surprisingly; therefore, the diagnosis of rare diseases is often delayed. Rare diseases affect likely 2-5% of the population. These diseases frequently have a hereditary premise, either as exceptional latent conditions or as the aftereffect of "de novo" hereditary changes not present in either parent. They can take shifted and regularly incapacitating structure.

Track 5: Challenges in Rare Diseases Treatment

There are approximately 7,000 rare diseases, which from a regulatory outlook are defined as those diseases where there are less than 200,000 patients in the US or that affect no more than 5 in 10,000 of the general population in the EU. Orphan drugs are medicinal products envisioned for diagnosis, prevention, and treatment of deadly rare diseases. They are "orphans" because the pharmaceutical industry has very little interest under normal market conditions in developing and marketing drugs used for only a very small number of patients who are suffering from rare conditions.

Track 6: Rare Skin Diseases

Millions of people live with at least one skin condition. Skin diseases affect one in four Americans. We are probably familiar with the more common ones like acne, eczema, and rosacea. In fact, there’s a good chance you may have one of them yourself. There’s also a wide range of rarer skin conditions that you may not know about. They can range from mild to life threatening. In some cases, they can affect the quality of life of those who develop them.

Track 7: Orphan Drugs- development trends and strategies

The advancement of Pharma Industries has eased back as of late due to numerous reasons such as patent generic competition, expiries, drying pipelines, and increasingly stringent regulatory guidelines. Many useful drugs will lose their exclusivity in next 5 years. Accordingly, the present financial circumstance and the huge generic competition shifted the focus of pharmaceutical companies from the essential medicines to the new business model — niche busters, also called orphan drugs.       

Track 8: Clinical Research on Orphan Drugs

A clinical preliminary is a therapeutic report led to test the impacts of another or effectively existing medication, of a biological treatment or of a medical device that might treat or curb a disease already identified. The main goal of a clinical trial is to compare two or several groups of subjects, by using two or several treatments in order to determine the efficacy of a drug or of a biological treatment. Clinical preliminaries are cautiously and morally led so as to secure patients against undesirable unfavorable responses and to allow collection and accurate analysis of the information concerning the disease.

Track 9: Rare Diseases in Children

Most of the rare diseases appear in early stage of life, about 30% of children with rare diseases will die before reaching their fifth birthday. With only three diagnosed patients in 27 years, ribose-5-phosphate isomerase deficiency is considered the rarest known genetic disease. Most of the rare diseases are genetic and are present throughout the person's entire life, even if symptoms do not appear immediately.

Track 10: Orphan Drugs and Ethical Issues

This track plots the ethical quandary of funding orphan drug research and development. Until this point in time, moral parts of need setting for look into subsidizing have not been an issue of discourse in the bioethics debate. Clashing good commitments of value and distributive equity seem to request altogether different degrees of funding for orphan drug research. The two kinds of orphan disease, rare diseases, and tropical sicknesses, in any case, present altogether different moral difficulties to inquiries concerning the allotment of research reserves. The quandary is dissected considering utilitarian and rights-based speculations of equity and good commitments of non-deserting and an expert commitment to propel restorative science. The limitations of standard economic evaluation tools and other priority setting tools used to inform health policy decision makers on research funding decisions are outlined

Track 11: Rare Diseases in Cancer

Rare cancers caused by simple genetic mutations and common cancers tend to be caused by a complex set of genetic and epigenetic aberrations that continually grow in number as the tumor develops. A cancer is considered to be rare if-

 

1. It starts in an uncommon place in the body,

2. The cancer is an unusual type and it may need special treatment,

3. It is not one of the common types of cancer.

Track 12: Case reports on Rare Diseases

According to Medicine: A case report is a definite report of the symptoms, signs, diagnosis, treatment, and follow-up of an individual patient. Case reports may contain a segment profile of the patient, yet for the most part portray a strange or novel event. Some case reports likewise contain a writing survey of other detailed cases. On the off chance that various case reports show something comparative, the subsequent stage may be a case-control study to decide whether there is a connection between the applicable factors.