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Rare Diseases Congress- 2019

About Conference


We are happy to announce this, with the success of previous Rare Diseases Congress-2018, we are delighted to invite all the participants all over the globe to attend “9th World Congress on Rare Diseases and Orphan Drugs”, will be held during June 17-18, 2019 in Rome, Italy which includes important keynote presentations, Oral talks, Poster presentations and Exhibitions in the field of Rare Diseases and Orphan Drugs

The main aim of this Congress is to unite the international rare disease community to take a fresh look at the key topics and challenges in Rare Diseases research. Rare Diseases Congress 2019 is a combination of the expert Rare Diseases researchers, Medical Professionals, pharmacologists, industrialists, Pharmaceutical Companies and other legendary professionals in the field of Rare and Orphan Diseases. At Rare Diseases Congress 2019 we are ready with the Novel Research and research techniques in the field of Rare Infectious Diseases.

We Organizes 1000+ Global Events Every Year across USA, Europe & Asia with support from 1000 more scientific societies and Publishes 700+ Open access journals which contains over 100000 eminent personalities, reputed scientists as editorial board and organizing committee members. 

Rare diseases refer to those medical abnormalities which affect a very least number of the population. They are characterised by a wide variety of disorders and symptoms that differs not only from disease to disease but also from individual to individual patient suffering from the same disease. About 80% of these disorders have recognized genetic origins while the others are the result of infections, allergies and environmental causes. Rare Diseases conventions the academic and long-term research developments can be discussed enabling a comparison between different theories, concepts and approaches

Target Audience:

Pathologists

Infectious Diseases Specialists

Pharmacists

Epidemiologists

Health Care Professionals

Microbiologists

Bacteriologists

Virologists

Parasitologists

Mycologists

Researchers

Training Institutes

Pharmaceutical Companies

Rare Diseases Associations

Orphan Drugs Companies

Why to attend?

Rare Diseases and Orphan Drugs conference represents serious uncommon/ unrelated complications that have diverse impacts on people, public health, and economies. These diseases are responsible for rising healthcare costs, lost productivity, and long-term disability. This conference will feature a global audience of scientific leaders, academia and health care professionals, and Orphan Drugs experts who are going to discuss today’s emerging treatments and diagnostics. By bringing together the Rare Diseases community, presenting the latest developments, and identifying unmet treatment needs, Rare Diseases Congress-2019 hopes to accelerate research and drug development in Rare Diseases, improving patient outcomes. It is designed to provide an educational forum that stimulates clinical, research, government and patient communities to create a coordinated and comprehensive approach for advancing the implementation of research, clinical care, care delivery coordination and other public health interventions critical to improved outcomes in Rare Diseases and Orphan Drugs research.

For Scientific Sessions please go through the link: 

http://rarediseases.conferenceseries.com/europe/scientific-program

For Abstract Submission please go through the link:

http://rarediseases.conferenceseries.com/europe/abstract-submission.php

 

 

 

Market Analysis

Summary

Rare Disease is an orphan disease that affects a small percentage of the population all over the world. An orphan disease is rare diseases with a lack of a market large enough to gain support and sources for discovering treatments for it, with advances to research in orphan diseases advantageous conditions to creating and selling such treatments. Rare diseases are genetic and are present throughout the entire life of individual suffering, even if symptoms do not immediately appear. Many rare diseases seems to appear early in life, and about 30% of children affecting with rare pediatric diseases will die before getting 5 year old.

Rare diseases are generally genetic and so they chronic. It is estimates that at least 80% of them have identified genetic origins. Other rare diseases are the result of infections and allergies or it may be due to degenerative and proliferative causes and discovering the well advanced treatments in Rare Diseases and Orphan Drugs.

Importance & Scope

There are more than 6000 rare diseases. Many rare diseases are diagnosed at the age of childhood, making diagnostic awareness and knowledge on treatment and care particularly important for paediatricians

The rare diseases are so rare that rarity can lead to several problems including: difficulties in obtaining timely, accurate diagnoses; lack of experienced healthcare supervisors; useful, reliable and timely information may be difficult to retrieve; research activities are very less; developing new pharmaceuticals may not be economically feasible; treatments are sometimes very expensive; and in developing countries, the problems are compounded by other resource limitations.

Rare Diseases Congress 2019 is emerging up with an exciting and high knowledge gainig conference program this year also which includes plenary lectures, symposia, workshops on a variety of topics, poster presentations and various programs for participants from throughout the world. We invite you to join us at the Rare Diseases and Orphan Drug Congress 2019 in Italy where you will be sure to have a meaningful experience with scholars from around the world. The Rare Diseases Conference aims to extend the reach, impact and exchange of scholarly ideas through innovative technology, determined commitment and exceptional service. It aims to provide international collaboration and exchange of comprehensive and cutting-edge information on basic, translational, and applied clinical research on rare diseases and Orphan Drugs

Why Rome??

Italy was one of the first Member States (MS) in the European Union (EU) to regulate the field of Rare Diseases (RD). Since 2001, many initiatives were carried out at central and regional level. The development of a national plan or strategy for RD stems. Rome serves as second largest city in Italy, with Tarentum as the largest one. Being the capital of Italy, Rome heads all the principal institutions of the nation, including the Presidency of the Republic, the government the Parliament, the main judicial Courts, and the diplomatic representatives of all the countries for the states of Italy and Vatican City.Rome has the status of a global city. Being ranked as the 14th most visited city in the world, in European Union its 3rd most visited one, and the very popular tourist attraction in Italy. Rome is the place of several specialized agencies of the United Nations, such as the Food and Agriculture Organization (FAO), the World Food Programme (WFP) and the International Fund for Agricultural Development (IFAD).

Members Associated with Rare Diseases and Orphan Drugs Research

Rare Diseases Congress 2019 is to foster international research collaboration and investment in the field of rare diseases, with the aims to contribute to the development of 200 new therapies and the means to diagnose most rare diseases by the year 2020.  Rare Diseases Congress has the ultimate goal to improve diagnosis and therapy for patients affected by rare diseases. Briefly, the composition of members reflected the implementation of the Rare Diseases and Orphan Drugs concept from the high level discussions between the United States of America National Institutes of Health (NIH) and the European Commission (EC), recognizing the need to bring together research funders, policy makers, regulators, patient organizations, premier research institutes, and industry to represent almost 350 million people living with a rare diseases and treatment with Orphan Drugs.

Major Associations Associated with Rare Diseases Research

Alzheimer's disease Organizations

Ann & Robert H. Lurie Children’s Hospital

Birmingham children’s Hospital

Boston Children’s Hospital

Canadian Organization for Rare Diseases

Chicago Rare Disease Foundation

Children’s hospital of Pittsburgh

Comer Children’s Hospital – University of Chicago

Cystic Fibrosis Foundation

European Union Committee of Experts on Rare Diseases

EURORDIS Rare Diseases Europe

EveryLife Foundation for Rare Diseases

Global Genes Allies in Rare Diseases

Guardian Hands Foundation

Hospitals Associated with Rare Diseases Research

IRDR Intractable & Rare Diseases Research

Japan Patient Association

Multiple Myeloma Research Foundation

National Alliances for Rare Diseases

National Institute of Health (NIH)

National Organization for Rare Diseases

NDC Medicine

Organization for Rare Diseases India (ORDI)

Orphan Europe

Philippine Society for Orphan Disorders

Rare Disease UK

Rare Diseases or Syndromes and Clinical Societies

Rare Diseases Foundation, Iran

Rare Diseases Patient Association Funding

Rare Diseases South Africa

Rare Diseases Translational Research Collaboration

Rare Disorders Society Singapore

RARE Foundation Alliance

Royal Society of Medicine

Short Bowel Syndrome Foundation

Students4RareDiseases

The Asia-Pacific Alliance of Rare Disease Organisations

The Boler-Parseghian Center for Rare & Neglected Diseases

The Boler-Parseghian Center for Rare & Neglected Diseases

The Children’s Hospital of Philadelphia

The Every Life Foundation for Rare Diseases

The Genetic and Rare Disorders Organization

The Greek Alliance for Rare Diseases

The Manton Center for Orphan Disease Research

U.S. Food and Drug Administration

UCLA Health

US hospital for Rare Disease Research

 

Major Universities Associated with Rare Diseases Research

Anahuac University North Campus, Mexico

Australian National University, Australia

Benha University, Egypt

Birmingham City University, UK

Center for Clinical Pharmacology, Belgium

Center for Rare Neurological Diseases, USA

Charles Darwin University Casoria Australia

Columbia University Medical Center, United States

Columbia University, USA

Curtin University Bentley, Australia

Dar Al Uloom University, Saudi Arabia

Duke University, USA

Emory University, USA

GMEC, The Global Medical Excellence Cluster

Guangzhou Medical University, China

Harvard University, United States

Harvard University, USA

Imperial College London, United Kingdom

Infection Control University

Iqbal Chest Centre, Bangladesh

John Hopkins University, USA

Johns Hopkins University, United States

Karolinska University, Sweden

Kindai University, Japan

King Saud Bin Abdulaziz University, Saudi Arabia

King's College London, UK

Kumamoto University, Japan

Linnaeus University, Sweden

Macquarie University, Australia

Mayo Clinic College of Medicine, USA

McGill university   Montréal, Canada

McMaster University, Canada

Medi7 Bentleigh, Australia

Murdoch University   Murdoch, Australia

National Institutes of Health, USA

Newcastle University, Australia

Northwestern University, Qatar

Osaka University, Japan

Oxford University, UK

Philip Morris International R&D, Switzerland

Pompeu Fabra University, Spain

Queen Mary University, UK

Radboud University Medical Center, Netherlands

Rare Genomics Institute, USA

Research Institute of Hospital del Mar, Spain

Rizzoli Orthopedic Institute, Italy

Samsung Medical Center, South Korea

St George’s University of London, UK

St. George Hospital, Australia

Stanford University, USA

Tasmanian Health Service, Australia

The Chest & Heart Association of Bangladesh, Bangladesh

The Fourth Hospital of Harbin Medical University, China

The Jikei University School of Medicine, Japan

The Third Affiliated Hospital of Guangzhou Medical University, China

The University of Newcastle, Australia

Tufts university, United States

United Hospital, Bangladesh

University College London, UK

University of British Columbia, Canada

University of Buffalo, United States

University of California Los Angeles, United States

University of California, USA

University of Cambridge, USA

University of Canberra Bruce, Australia

University of Celiac Disease Center, USA

University of Chicago Medicine, USA

University of Colorado, USA

University of Groningen, Netherlands

University of Lincoln, UK

University of London Imperial College of Science Technology And Medicine, UK

University of Maastricht, Netherlands

University of Maryland Medical Center, Australia

University of Maryland Medical Center, United States

University of Melbourne, Australia

University of Minnesota, United States

University of Newcastle, Australia

University of Pennsylvania, USA

University of Pittsburgh Study

University of Pittsburgh, USA

University of Queensland, Australia

University of Tasmania, Australia

University of Toronto, Canada

University of Valencia, Spain

University of Washington, USA

University of Zurich, Switzerland

University-of-the-sunshine-coast, Australia

Weill Cornell Medical College, Qatar

Wits University, South Africa

Yale University School of Medicine, USA

Yonsei University, South Korea

Major Companies Associated with Rare Diseases and Orphan Drugs

700thespians Limited, UK

Abbott Laboratories, UK

Actavis, USA

Aegerion Pharmaceuticals, Japan

Alexion Pharmaceuticals Inc, Switzerland

Almirall, Spain

Amgen, USA

Amicus Therapeutics, USA

Amphastar Pharmaceuticals, Inc

Araim Pharmaceuticals

Astellas Pharma US

AstraZeneca, Switzerland

AstraZeneca, UK

Baxter International Deerfield

Bayer HealthCare, Germany

Bayer, Germany

Beacon Pharmaceuticals, Bangladesh

Biotie Therapies Corp, Finland

Bioxyne Limited, Australia

Boehringer Ingelheim, Germany

Celgene, Switzerland

Centrapharm Ltd, UK

Chiesi Pharmaceutical, Italy

Clalit Health Service, Israel

Cohero Health, USA

Daiichi Sankyo Europe, Germany

Daiichi Sankyo, Japan

Dohmen Life Science

Elma Reseach, UK

European Medicines Agency

Focus Scientific Research Center (FSRC), phamax, India

Forest Laboratories, USA

Gecko Health, USA

Generics (UK) Ltd, UK

Genus Oncology Vernon Hills, USA

Genzyme, USA

Gilead sciences, USA

GlaxoSmithKline, UK

Global Data, UK

Gsk, London

Hormosan Pharma, Germany

Hormosan Pharma, Germany

Ikris Pharma Network Pvt Ltd, India

Ikris Pharma Network Pvt Ltd, India

Kissei Pharmaceutical Co., Ltd, Japan

Kyowa Hakko Kirin Co. Ltd, Japan

Lallemand Pharma, Switzerland

Life Arc, UK

Marathon Pharmaceuticals

Merck & Co, USA

Millennium Pharmaceuticals

Napp Pharmaceuticals Ltd, UK

Novartis, Switzerland

NPS Pharmaceuticals

Octapharma, USA

Onyx Pharmaceuticals, USA

Otsuka Holdings Co., Ltd, Japan

Panmira Pharmaceuticals, LLC, USA

Pearl Therapeutics, Inc

Pfizer, USA

PharmaMar, USA

Prosensa, Netherlands

PT Boehringer Ingelheim, Indonesia

Queensland Respiratory Laboratory Pty. Ltd, Australia

Ranbaxy Laboratories Limited

Raptor Therapeutics

Roche, Switzerland

Samsung Medical Center, South Korea

Sanofi, France

Sarepta Therapeutics, USA

Sigma-Tau Pharmaceuticals, Italy

Sunovion Pharmaceuticals, USA

Swedish Orphan Biovitrum AB, Sweden

Takeda, Japan

TriStem Corp Ltd, UK

Vertex Pharmaceuticals, USA

ViroPharma

ViroPharma, USA

Visionary Pharmaceuticals, Inc, USA

Yungjin Pharm Ind. Co., Ltd, South Korea

Market Research Growth on Rare Diseases and Orphan Drugs

Nearly there are 7,000 different types of rare diseases and disorders it has estimated that 30 million people in the United States, 30 million in Europe and 350 million people over the globe suffer from rare disease. Four by fifth of rare diseases are genetic in origin, among them affected individuals 50% of them will be children. The rare diseases are distributed in such a way that four fifth of the cases accounted by some 350 rare diseases. About only 5% of rare diseases are having approved drug treatment with only 326 new drugs being approved from the FDA and brought in to the market.

In 2018, orphan drug sales were of the order of 93 billion. Orphan drugs, represented 35% of the industry’s new drug approvals. The genetic diseases are subdivided by therapeutic area, which was leading the global market in the past will show similar traction in the coming next eight years. This segment is anticipated to be valued at US$ 56,241 by the end of 2025.

Sessions/Tracks

1. Rare Diseases in Neurology

Neurological disorders are the diseases associated with the spine, brain and the nerves that connect them. It effects the body nervous system. Reflex Sympathetic Dystrophy Syndrome, Aicardi-Goutieres syndrome, Aicardi syndrome are few rare 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 are 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.

2. Rare Mental and Behavioral disorders

Abnormality is also called as dysfunctional behavior which refers to the behavioral characteristic assigned to those with conditions regarded as rare or dysfunctional. A Behavior is considered to be abnormal when it is atypical or out of the normal or usual,it consists of unpleasant behavior, and results in deterioration in the individual's functioning. Abnormality is one, which is considered as different from specific ethical social and cultural expectations. These expectations are broadly dependent on age, gender, traditional and societal categorizations. The definition of abnormal behavior is an often debated issue in abnormal psychology because of these subjective variables.

3. Rare Pediatric Diseases

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.

4. Immunological Rare Diseases

The immune system is delegated to defend the body from attacks from outside or inside. Many diseases can affect immune system reducing its ability to defend self or inducing an abnormal response against external or internal antigens. Rare diseases affecting immune system present some issue in common with other rare diseases and some peculiarities due to the huge variability in the disease's expression. However, a correct estimation of the epidemiology of rare disorders is necessary for evaluating the prognosis and the responses to new therapies, for planning proper public health services, and finally to establish fair and sustainable prices for innovative medicines. The immune system is delegated to defend the body from attacks from outside or inside. Many diseases can affect immune system reducing its ability to defend self or inducing an abnormal response against external or internal antigens. 

5. Rare Diseases in Nephrology

Many rare diseases affect the kidneys and the urinary system, some limited to the urinary system itself (eg, membranous nephropathy), and some part of a condition (eg, Alport syndrome) that  causes additional symptoms in other organ systems. With varying degrees of severity, these diseases can affect different parts of the urinary system (kidney glomerulus, kidney tubules, urinary tract, etc) causing numerous symptoms.Several of these diseases occur in the context of inflammation and autoimmune disease (eg, Goodpasture syndrome). Many rare types of vasculitis can also cause major kidney problems (eg, Henoch-Schönlein purpura nephritis).Disorders of kidney development, kidney metabolism, or substance transport within the kidney also exist. Some are directly heritable diseases caused by genetic defects (eg, cystinuria). Rare  diseases can cause acute kidney damage (which may be repairable), but more often result in chronic kidney disease.

6. Rare Skin Diseases

Poor understanding of the explanation of the planned indication thanks to few data-based studies finding out malady progression, heterogeneous patient populations with variable phenotypes and clinical courses, geographic dispersion of patients and investigators, restrictive uncertainties, and lack of previous clinical studies to determine a template for study execution, In rare malady trials, the necessity to recruit and retain patients whereas adhering to exceptional standards of care influences each call. The protocol should account for the vulnerability of the patient population and address moral concerns, significantly if the study style mandates ending of current medical aid thought-about essential for patient support. Eligibility criteria invariably influence the quantity of accessible subjects, and if unnaturally forced, scale back the chance of creating clinical trials info from that proof of effectiveness and safety

7. Rare Oncology

The many different types of rare oncological diseases include tumors of the body and hematological disorders of the blood. Although individually these malignancies are rare, as a group, they represent a significant majority of people with cancer.Certain characteristics of these malignancies differentiate them from more common types of cancer. Often, the cancer begins in a type of cell that only rarely becomes cancerous. This can happen in organs that are otherwise common sites of cancer growth. For example, although breast cancer is not a rare disease, angiosarcoma of the breast, which arises in the inner lining of blood vessels, is a rare cancer.

8. Rare Diseases of Lymphatic System

These diseases occur very rarely, consisting of fluid-filled channels they are non-malignant masses or spaces thought to be caused by the abnormal development of the lymphatic system. These formations are usually detectable at birth or after two years of age. Lymphatic malformations can affect any part of the body (except the brain), but they most commonly affect the head and neck. When recognized at birth that is congenital, lymphatic malformations tend to be soft, spongy, non-tender masses. The most common symptoms and lymphatic malformations severity differs depending  upon the size and specific location of the malformation. Some lymphatic malformations can be massive or huge.Lymphatic malformations can potentially cause functional impairment of nearby structures or organs and disfigurement of affected areas Irrespective of size.

9. Rare Nutritional and Metabolic Diseases

Metabolism is the chemical process which our body uses to transform the food we eat into the form energy or fuel that keeps us alive and energetic too. When the metabolism processess fails a metabolic disorder causes and results in the body to have either too high or too little of the essential materials needed to stay healthy. Our body is very sensitive to mistakes or errors in metabolism. The body must have amino acids and many types of proteins to perform all of its metabolic functions.  Calcium, potassium, and sodium ions are utilized  by our brain to generate electrical impulses and to send signals, and also lipids and fatty acids to maintain a healthy nervous system

10. Rare Gynecological and Obstetrical Diseases

Gestational Diseases (GTD) may be defined as bunch of rare diseases that develop within the womb throughout an abnormal physiological condition. It happens once when abnormal cells or tumors grow inside the tissues that are square measure shaped following conception that may unremarkably surround the embryo and transform the placenta. Redness is caused by Acute venereal disease which might result in the Fitz-Hugh-Curtis syndrome (perihepatitis that causes higher right quadrant pain). Infection may become chronic, which are  characterised by intermittent exacerbations and remissions

 

11. Treatment and Advanced Therapies options for Rare Diseases

Diagnosis and the treatment of a rare disease become complex because of the circumstances that numerous health care providers may have restricted involvement with the identification of the particular disease condition with the diagnosis of rare diseases. Additionally, analysis before manifestation onset or diagnosis early stage in the disease can be challenging. Biopharmaceutical experts in the field have utilized new advances and the developing investigative comprehension of many rare diseases to develop and diagnose ground breaking therapies over the last 10 years. In 2015 alone, about half (47%) of novel latest drug approvals were for rare diseases. Disease specific therapies need to be developed for productive results in treatment of Rare Diseases.

12. Orphan Drugs Potentiality

An orphan drug is referred medicinal agent which has been developed to treat a rare medical illness or situation, the condition itself being defined to as a rare disease. Rare disease, also known as an "orphan disease", is any type of disease which affects a small percentage of the population. Majority of rare diseases are genetic, and hence are present throughout the individual's entire life, even if symptoms doesn’t appear  immediately. Many rare diseases appear early in life, and about 30 % of children with rare diseases will die before getting 5 year old. With only a solo diagnosed patient, ribose-5-phosphate isomerase deficiency is presently considered to be the rarest genetic disease. No single cut-offs number has been decided upon for which a disease is considered rare. A illness may be considered rare in one part of the world, or in a particular group of people, but still be common in another country or group of people. According to a research  over 55 million people are estimated to suffer from a rare disease in Europe and in the US, global estimates are between 5000 to 7000 rare diseases and new rare diseases are revealed every week and for many treatment is not available.

13. Clinical case study on Rare Diseases

A clinical test is an experimentation 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:

14. Rare Diseases of Bloodstream

Breakdown in regular process of blood cell growth and development leads to the cell types production in abnormally large quantities or in abnormal form. Some rare blood disorders especially those that are gene-based are easy to identify with a simple test, while others require substantial analyst work to successfully diagnose. Due to the large number of blood conditions common and uncommon, haematologists tend to be skilled investigators because they need to be specialists in differential diagnosis. This leads to blood cancers or other blood disorders. The most common blood cancers includes leukemia, lymphomas, and multiple myeloma. There are few common blood disorders also. These include various myeloproliferative neoplasms, histiocytosis, and paroxysmal nocturnal hemoglobinuria.

15. Rare Diseases of Endocrine System

Even though numerous endocrine system illnesses such as diabetes, thyroid, and osteoporosis are among the utmost common and well known medical conditions, some patients who have been referred to an endocrinologist will require treatment for a rare disease. Number of these rare diseases are dangerous and lead to myriad sequelae and secondary diseases. Gland-based hormonal imbalances resulting in developmental abnormalities and related complications are among these diseases ,weight regulation issues and physical abnormalities  and frequent carcinogenic and non-carcinogenic endocrine-related tumor disorders (eg, multiple endocrine neoplasia, neuroendocrine cancer adrenocortical carcinoma, papillary thyroid carcinoma, primary thyroid lymphoma). Rare endocrine diseases can potentially impact any organ system, however.

 

16. Rare Musculoskeletal Diseases

A wide range of rare conditions involve the musculoskeletal system.Some, like Duchenne muscular dystrophy, arise from genetic problems in the muscles themselves, while others involve the nerves (eg, Charcot-Marie-Tooth disease), motor neurons (eg, amyotrophic lateral sclerosis [ALS]), or the bones (eg, osteogenesis imperfecta). Problems in other part of the body—such as mitochondrial disease or a metabolic disease like carnitine deficiency—can also lead to major problems with these systems.Many individuals are familiar with some of these conditions, thanks ,in part, to powerful advocacy groups. Nonetheless, treatment options remain limited—although extensive research may soon change that.

 

17. Rare Hereditary Diseases

Researchers have made dramatic inroads into the study of polygenic and other complex human diseases, due in large part to knowledge of the human genome sequence, the generation of widespread markers of genetic variation, and the development of new technologies that allow investigators to associate disease phenotypes with genetic loci. Although polygenic diseases are more common than single-gene disorders, studies of monogenic diseases provide an invaluable opportunity to learn about underlying molecular mechanisms, thereby contributing a great deal to our understanding of all forms of genetic disease.

Past Conference Report

Rare Diseases Congress-2018

Rare Diseases Congress-2018 Past Conference Report

4th World Congress on Rare Diseases and Orphan Drugs was held in June 11-12, 2018 in Dublin, Ireland. The conference was marked with the presence of Editorial Board Members of supporting journals, Scientists, Young and Brilliant Researchers, Business Delegates and talented student communities representing more than 20 countries, who made this conference fruitful and productive. This conference was based on the theme, Connect together for better care and innovations in rare disorders without borders.

Rare Diseases Congress-2018 covered the following scientific sessions and discussions:

  • Rare Genetic Diseases
  • Pediatric Rare Diseases
  • Rare Hereditary Diseases
  • Diagnosis and Treatment for Rare Diseases
  • Clinical Case Studies on Rare Diseases
  • Current Rare Diseases Research
  • Orphan Drugs Market Research
  • Rare Diseases of Sexual Health
  • Scope of Orphan Drugs
  • Rare Diseases in Neurology

We are thankful to our below honorable guests for their generous support and suggestions:

  • Laxminarayan Bhat, Reviva Pharmaceuticals Inc., USA
  • Gianluca Colella, Rizzoli Orthopaedic Institute, Italy

The conference proceedings were carried out through various scientific-sessions and plenary lectures of which the following topics were highlighted as Keynote presentations:

  • “Serotonergic targets in the treatment of pulmonary fibrosis (PF) and pulmonary arterial hypertension (PAH)” given by Laxminarayan Bhat, Reviva Pharmaceuticals Inc., USA
  • “Surgical treatment of early onset scoliosis associated with rare disease” given by Gianluca Colella, Rizzoli Orthopaedic Institute, Italy
  • “Clinical course and outcomes of critically ill patients with Middle East respiratory syndrome coronavirus infection” given by Abdulaziz Aldawood, King Saud Bin Abdulaziz University, Saudi Arabia

Conference Series LLC has taken the privilege of felicitating Rare Diseases Congress-2018 Organizing Committee, Editorial Board Members and Keynote Speakers who supported for the success of this event. The esteemed guests, keynote speakers, well-known researchers and delegates shared their innovative research and vast experience through their fabulous presentations at the podium of grand Rare Diseases Congress-2018. We are glad to inform that all accepted abstracts for the conference have been published in Conference Series LLC Journal of Genetic Syndromes & Gene Therapy as a special issue.

We are also obliged to various Delegate Experts, Company Representatives and other eminent personalities who supported the conference by facilitating active discussion forums. We sincerely thank the Organizing Committee Members for their gracious presence, support, and assistance. With the unique feedback from the conference, Conference Series LLC would like to announce the commencement of the "9th World Congress on Rare Diseases and Orphan Drugs” to be held during June 17-18, 2019 in Rome, Italy.

Let us meet again @ Rare Diseases Congress-2019

 

Past Reports  Gallery  

To Collaborate Scientific Professionals around the World

Conference Date June 17-18, 2019

For Sponsors & Exhibitors

[email protected]

Speaker Opportunity

Supported By

Journal of Genetic Syndromes & Gene Therapy Journal of Clinical Infectious Diseases & Practice Journal of Clinical Trials Journal of Neuroinfectious Diseases

All accepted abstracts will be published in respective Conference Series LLC LTD International Journals.

Abstracts will be provided with Digital Object Identifier by