6th Annual Congress on Rare Diseases and Orphan Drugs (CSE)

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Thu, Sep 23 08:00 AM 2021 - Fri, Sep 24 08:00 AM 2021
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, Toronto, , Canada,
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6th Annual Congress on Rare Diseases and Orphan Drugs


Rare Diseases 2020

On behalf of the organizing committee, we are pleased to announce that the 6th Annual Congress on Rare Diseases and Orphan Drugs (Rare Diseases 2020) will be held from September 23-24, 2020 in Toronto, Canada. Rare Diseases 2020 provides a premier interdisciplinary platform for researchers to present the latest research findings and describe emerging technologies, and directions in rare diseases and orphan drugs issues. The conference seeks to contribute to presenting novel research results in all aspects of Rare Diseases and Orphan Drugs.

The conference aims to bring together leading academic scientists, researchers and research scholars to exchange and share their experiences and research results about all aspects of rare diseases and orphan drugs. It also provides the premier interdisciplinary forum for scientists, professors, specialists, researchers, students and practitioners to present their latest research results, ideas, developments, and applications in all areas of rare diseases and orphan drugs. The conference will bring together leading academic scientists, researchers, Industrial delegates Healthcare Professionals and scholars in the domain of interest from around the world.

The conference's goal will to provide a scientific forum for all international prestige scholars around the world and enable the interactive exchange of state-of-the-art knowledge. The conference will focus on evidence-based benefits proven in clinical trials and scientific experiments.

Why to attend?

Join the Rare Diseases and Orphan Drugs Congress to keep up to date with the industry and to learn from our expert speaker panel, bringing you important new case studies and reports on this year’s relevant topics.

Learn more about global trends in rare diseases and its advances in the therapeutic and diagnostic market.
Discussing strategic win-win collaborations to help accelerate rare disease clinical drug development, and improve relationships between drug developers and patients to consider both parties’ interests
Hear more about how patient engagement by integrating the patient perspective into the drug development process through patient advocacy, patient-centric research, and patient groups
Learn about how the regulatory landscape for drug approvals is different between countries, and why it is important to keep informed about the regulations and guidelines of each region
Gain knowledge of different rare diseases and their unique challenges, as well as how treatment methods can be transferred to other rare diseases
Introducing cell and gene therapies to rare disease treatment - learn how gene therapy methods can improve the treatment of rare diseases and why it is becoming more commercially successful.

Market Analysis

Importance and Scope

A rare/orphan disease is outlined as a condition that affects fewer than 200,000 people. This definition was created by Congress within the Orphan Drug Act of 1983. There may be as many as 7,000 rare diseases and 70% of them have no form of treatment. Regulatory edges like longer market exclusivity, breakthrough designations, reduced fees and tax incentives are all encouraging investment. However, the marketing process and lifecycle for a rare disease drug are very different to a mass market product and require particular skills and knowledge.

Rare Diseases 2020 will be the best platform for all the doctors, researchers, renowned Scientists, research scholars, students who are working in this field across the globe under a single roof to exchange their knowledge related to Rare Diseases and Orphan Drugs. This international event is an effort to find an alternative for invasive imaging technique against rare diseases like Zellweger syndrome, Muscular Dystrophy, Alkaptonuria, Angelman syndrome, Prader-Willi syndrome, Tay Sachs disease, Fragile X syndrome, Gaucher disease as well as many others.

Conference Highlights

· Different types of Rare Diseases

· Clinical Research and Public Awareness

· Mystery Diagnosis of Rare Diseases

· Challenges in Rare Diseases Treatment

· Rare Infectious Diseases and Immune Deficiencies

· Rare Diseases in Cancer

· Rare Diseases in Aging

· Orphan Drugs- development trends and strategies

· Clinical Research on Orphan Drugs

· Orphan Drugs and Ethical Issues

· Future Hereditary of Rare Diseases and Orphan Drugs

· Patient organizations and their role in drug development or clinical research

Why attend?

In today's economic climate your business selections are as crucial as ever. 6th Annual Congress on Rare Diseases and Orphan Drugs (Rare Diseases 2020) allow you to maximise your time and marketing dollars whereas receiving immediate feedback on your new merchandises and services. Rare Diseases 2020 is organizing an outstanding Scientific Exhibition/Program and expects the world's leading specialists involved Rare Diseases and Orphan Drugs. Your company will benefit with outstanding exposure to the leaders in Rare Diseases and Orphan Drugs. Rare Diseases 2020 is a stimulating opportunity to showcase the new technology, the new products of your company, and/or the service your industry may offer to a wide-ranging international audience.

Target Audience

Join the Rare Diseases and Orphan Drugs congress to keep up to date with the industry and to learn from our expert speaker panel, bringing you important new case studies and reports on this year’s relevant topics.

· Directors, Board Members, Presidents, Vice Presidents, Deans and Head of the Departments

· Researchers, Scientists, Faculties, Students

· Rare Diseases Associations and Societies

· Medical Colleges

· Industrial delegates from Academia and Research

· Industrial professionals from biomedical companies and healthcare sectors

· Medical Devices Manufacturing Companies

· Drug Manufacturing Companies and Industries

· Laboratory Technicians and Diagnostic Companies

· Business Entrepreneurs and Industrialists


The scientific program will focus on current advances in the clinical research, clinical trials, drug discovery, drug development, drug treatment and orphan drug clinical trials with a particular focus on their role in maintaining the academic level in rare diseases and orphan drugs. In addition, mentored sessions for poster presenters will provide guidance from world leaders for our experts of the future.

Track 1: Different types of Rare Diseases

A rare disease is defined as a condition that affects less than 200,000 people. This explanation was formed by Congress in the Orphan Drug Act of 1983. There may be as many as 7,000 rare diseases. Only a few categories of rare diseases are tracked when a person is diagnosed. These include certain infectious diseases, birth defects, and cancers. It also comprises the diseases on state newborn screening tests. Because most rare diseases are not trailed, it is hard to determine the exact number of rare diseases. Most rare diseases are genetic, and thus are present throughout the person's intact life, even if symptoms do not immediately appear.

Track 2: Clinical Research and Public Awareness

Clinical research is a branch of healthcare science. The first step in confronting this challenge is regularly getting the community to think about participating in clinical research. People need to consider how they can help advance the prevention, diagnosis, and treatment of disease. It is never too early to consider contribution whether or not someone finally chooses to join a study.

Track 3: Mystery Diagnosis of Rare Diseases

Diagnostic error in medicine is common. 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 2-4% of the population. These diseases often have a genetic basis, either as uncommon recessive conditions or as the result of “de novo” genetic mutations not present in either parent. They can take varied and often debilitating form.

Track 4: Challenges in Rare Diseases Treatment

There are around 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 five in 10,000 of the general population in the EU. Orphan drugs are medicinal products envisioned for diagnosis, prevention, and treatment of life-threatening rare diseases. They are "orphans" because the pharmaceutical industry has little interest under normal market conditions in developing and marketing drugs intended for only a small number of patients suffering from very rare conditions.

Track 5: Rare Infectious Diseases and Immune Deficiencies

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

Track 6: 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 may need special treatment, 3. It is not one of the common types of cancer.

Track 7: Rare Diseases in Aging

Aging is a collection of degenerative changes that occur in organisms that lack the ability to perpetually regenerate. Age is a major risk factor for most common neurodegenerative diseases. Dementia becomes more common with age. The spectrum includes mild cognitive impairment, Alzheimer's disease, cerebrovascular disease, Parkinson's disease and Lou Gehrig's disease. Rare diseases provide much insight into the cellular processes that hasten the aging process.

Track 8: Orphan Drugs- development trends and strategies

The development of pharma industries has slowed in recent years because of many reasons such as patent expiries, generic competition, drying pipelines, and increasingly stringent regulatory guidelines. Many blockbuster drugs will lose their exclusivity in next 5 years. Therefore, the current economic situation plus 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 9: Clinical Research on Orphan Drugs

A clinical trial is a medical study conducted to test the effects of a new or already existing drug, 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 trials are carefully and ethically conducted in order to protect patients against unwanted adverse reactions and to allow collection and accurate analysis of the information concerning the disease.

Track 10: Orphan Drugs and Ethical Issues

This track outlines the moral dilemma of funding orphan drug research and development. To date, ethical aspects of priority setting for research funding have not been an issue of discussion in the bioethics debate. Conflicting moral obligations of beneficence and distributive justice appear to demand very different levels of funding for orphan drug research. The two types of orphan disease, rare diseases, and tropical diseases, however, present very different ethical challenges to questions about the allocation of research funds. The dilemma is analyzed considering utilitarian and rights-based theories of justice and moral obligations of non-abandonment and a professional obligation to advance medical 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: Future Hereditary of Rare Diseases and Orphan Drugs

Work over the past 25 years has resulted in the identification of genes responsible for ~50% of the estimated 7,000 rare monogenic diseases and it is predicted that most of the remaining disease-causing genes will be identified by the year 2020 and probably sooner. This marked acceleration is the result of dramatic improvements in DNA-sequencing technologies and the associated analyses. We examine the rapid maturation of rare-disease genetic analysis and successful strategies for gene identification. We highlight the impact of discovering rare-disease-causing genes, from clinical diagnostics to insights gained into biological mechanisms and common diseases. Last, we explore the increasing therapeutic opportunities and challenges that the resulting expansion of the 'Atlas' of human genetic pathology will bring.

Track 11: Patient organizations and their role in drug development or clinical research

Rare disease drug development could benefit substantially from increased patient engagement and input to enhance understanding of the key aspects of disease impact, ways to measure these impacts and patients' perspectives on the benefit-risk profile of potential therapies.

Track 12: Entrepreneurs Investment Meet

A platform aimed to connect Entrepreneurs, Proposers and the Investors worldwide. It's intended to create and facilitate the most optimized and viable meeting place for engaging people in global business discussions, evaluation and execution of promising business ideas. An investor could be able to find out the highest potential investment opportunities globally, which provide a good return on investment. For entrepreneurs, this would be an ideal place to find out suitable investors and partners to start and/or expand their business. Thus it is a perfect place to connect Entrepreneurs, Business Owners, Early Stage Companies and Established Corporates with National or International Investors, Corporate Investors, and Potential Business Partners.

Please contact the event manager Marilyn (marilyn.b.turner(at) ) below for:
- Multiple participant discounts
- Price quotations or visa invitation letters
- Payment by alternate channels (PayPal, check, Western Union, wire transfers etc)
- Event sponsorship

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