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RARE DISEASES MEET 2020

About conference

About conference:

We are very happy to announce this, with the good success of previous Rare Diseases Congress series, we are delighted to invite the participants all over the globe to attend “7th Global congress of Rare diseases and Orphan Drugs”, will be held during April 27-28, 2020 at Istanbul, Turkey. which includes prompt Keynote Presentations, Special Sessions, Workshops, Symposiums, Poster Presentations, Oral talks and Exhibitions in the field of Rare Diseases and Orphan Drugs.

Rare Diseases refer to those medical aberrations which affect a very least number of the population. They are characterized by a wide diversity 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 predictable genetic origins while the others are the result of infections, allergies, neurological related and environmental causes. A medicinal product developed for the treatment of a Rare Disease is an Orphan Drug. Under regular market environments the pharmaceutical industry has little attention in evolving and marketing products of Orphan Drugs proposed for only a small number of patients.

Why to attend?

World Rare Diseases 2020 is giving a worldwide stage to analysts that afford new insights into the concealed rare diseases. 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. 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 enhan​ced outcomes in Rare Diseases.

Target audience:

  • Dermatologists
  • Pharmaceutical Companies and Industries
  • Health care professionals
  •  Students
  • Scientists
  • Researchers
  •  Training institutes
  • Pharmacists
  •   Drug manufacturing companies
  • Bacteriologists
  • Virologists
  • Ophthalmologist
  • Microbiologists
  • Mycologists
  • Orphan Drugs Companies
  • Students
  • Patient Groups
  • Cardiologists
  • Medical Devices Manufacturing Companies
  • Laboratory Technicians and Diagnostic Companies
  •  Anesthesiologists
  • Neurologists
  • Parasitologists
  •  Business Entrepreneurs and Industrialists
  • Epidemiologists
  • Rare disease associations
  • Infectious Diseases Specialists
  • Pathologists

 

 

 

 

Sessions/Tracks

Track 1: Orphan diseases and public health

The field of rare diseases is complex and heterogeneous and suffers from a deficit of scientific and medical knowledge .The landscape of rare disease is constant evolving the new rare diseases and conditions being reported and identified regularly in medical literature. Apart from a few rare diseases  where significant progress has been made, still the field is nascent stage. For a long time, doctors, researchers and policy makers were unaware of rare diseases and until very recently no real research or public health policy concerning issues related to the field .This posses challenges in development of comprehended policy on rare diseases. Nevertheless, it is important to take step, in the short as well as long term, with the objective of talking rare diseases in a comprehensive and holistic manner.

Track 2: Allergic and Immunologic Disorders

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.

Track 3: Pediatric Rare 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.

Track 4: Infectious Diseases

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.

Track 5: Clinical Trials and Treatment

 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.

Track 6:  Rare Neurological Disorders

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.

Track 7: Neglected Rare Tropical Diseases

  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.


Track 8: Gene Therapy for Rare Diseases

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.

Track 9: Hematologic Disorders

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.

Track 10 : Rare Obesity and Metabolic Disorders

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.

 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.

 

Market Analysis

Rare Disease Treatment Market size is experience significant growth from 2019 to 2025. Increasing prevalence of rare diseases across the world will stimulate rare disease treatment market growth in the coming years. According to the National Institute of Health, approximately 30 million Americans (10% of the total population) suffer from one of the 7,000 rare diseases. Also, 50 percent of patients affected by rare disease are children and only 5 percent of rare diseases have treatment. Additionally, rising investment in rare diseases treatment categories and introduction of new drugs will boost the market growth in the coming years. For instance, more than 560 drugs are in development for the treatment of rare disease. Therapies in development include treatment for amyotrophic lateral sclerosis, multiple myeloma, cystic fibrosis and enzyme deficiency disorders. Rising government initiatives for spreading awareness regarding rare diseases along with presence of suitable healthcare infrastructure in developed and developing economies will drive the industry growth. For instance, the Orphan Drug Act 1983 was introduced by the U.S. to facilitate the development of orphan drugs for the treatment of rare diseases such as Amyotrophic Lateral Sclerosis (ALS), cystic fibrosis, muscular dystrophy, etc. Orphan Drug Act and two other laws The Best Pharmaceutical for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) were introduced to encourage the development of medicines for pediatric rare diseases. Additionally, favorable reimbursement policies and regulations for approval of drugs will escalate the growth of rare disease treatment market in the coming years. However, lack of trained personals and challenges faced by pharmaceutical companies during the time of drug development are major limiting factors for the market growth.
 

Past Conference Report

Rare Diseases Meet 2019

ME Conferences had successfully hosted the "6th International Conference on Rare Diseases and Orphan Drug" during June 17-18, 2019 at Dubai, UAE on the theme of  Challenges & opportunities in elimination of Rare diseases & orphan drug development.

The analysis of the conference evaluation forms shows that the event was very well received as a valuable and stimulating. The majority of delegates who completed the conference evaluation forms rated the conference as a whole as “excellent” or “very good”. The conference was palmy in transfer along famous speakers from varied supposed organizations and their preponderant talks enlightened the gathering.

The pragmatic meet organized by our management received a generous response from the domain, gifted Keynote Speakers, Speakers, Young Researchers, Students & Business Delegates who attended from different parts of the world created their valuable contribution to form it as a successful event. The conference was marked with the presence of eminent Speakers, Young Researchers, Students & Business Delegates driving the congress into the way of accomplishment with animating keynote appears.

The conference was initiated with a warm welcome note from our Honourable Guests & followed by Keynote sessions. We would like to convey the great appreciation to Dr. Ayman Noreddin from University of Sharjah, UAE & Dr. Wafaa Ahmed Zahran from Menoufia University, Egypt.

We are very overwhelmed by their presence and generous response.

Rare Diseases Meet 2019 Organizing Committee extends its gratitude and congratulates the honourable moderators of the conference Dr. Nilanjana Ghosh, North Bengal Medical College, India and Dr. Ruchika Butola, Rajiv Gandhi Super Speciality Hospital, India who contributed plenty for the graceful functioning of this event.

We have taken a privilege of felicitating our keynote Speakers:

  • Ayman Noreddin | University of Sharjah | UAE
  • Robert O Young | PH Miracle Centre | USA
  • Mohamad Miqdady | Sheikh Khalifa Medical CityUAE
  • Galina Migalko | Universal Medical Imaging Group | USA

Who shared their innovative researches and immense experience through their marvelous presentations during the conference.

The eminent personalities at the conference were

  • Dr. Sivasankari Murugan | Meenakshi Medical College Hospital and Research Institute | India
  • Ms. Joyvonne Kwamboka Ombega | Rare and Chronic Diseases Foundation | Kenya
  • Dr. Raimundo Dos Santos | Hospital Nacional Guido Valadares | India
  • Ms. Prashanthi Rayapati | Lynbrook High School | USA
  • And many more

Last but not the least; we are obliged to various delegate experts, Company representatives, eminent personalities who supported the Conference by facilitating active discussion forums. We would like to convey our sincere gratitude to all the supporters from Editorial Board Members of our Open Access Journals, Keynote Speakers, Honourable guests, Valuable Speakers, Students, delegates and special thank s to Media Partners  for their promotions to make this event a successful one.

With the unique feedbacks from the Conference Rare Diseases Meet 2019, we would like to announce the commencement of the 7th Global Congress on Rare Diseases and Orphan Drug to be held during April 27-28, 2020 in Istanbul, Turkey.

Mark your calendars for the upcoming Conference; we are hopeful to see you soon!

More Info:  https://rarediseases-orphan.conferenceseries.com/

If you are interested!

Email us at: rare-diseases@memeetings.com

HOPE TO MEET YOU ALL IN ISTANBUL, TURKEY

 


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Conference Date April 27-28, 2020

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Keytopics

  • Aarskog Syndrome
  • Achalasia
  • Achondroplasia Imaging
  • Achondroplasia Imaging
  • Acidemia Isovaleric
  • Acoustic Neuroma
  • Addison's Disease
  • African Iron Overload
  • Alkaptonuria
  • Ankylosing Spondylitis
  • Anorectal Melanoma
  • Canavan Disease
  • Cancer Treatment
  • Cancer Treatment
  • Challenges In Rare Diseases Treatment
  • Challenges In The Economic Evaluation Of Orphan Drugs
  • Childhood
  • Chordoma
  • Chronic Disease
  • Clinical And Emergency Guidelines
  • Clinical Trials
  • Clinical Trials
  • Clinical Trials
  • Congenital Adrenal Hyperplasia
  • Data Architechure Of Rare Diseases
  • Diagnostics
  • Diastrophic Dysplasia
  • Disease Specic Groups
  • Donath-Landsteiner Hemolytic Anemia
  • Dravet Syndrome
  • Drug Discovery
  • Drug Discovery
  • Fabry Disease
  • Friedreich's Ataxia
  • Gastrointestinal Stromal Tumours
  • Genetic Disorders
  • Genetic Disorders
  • Genetic Disorders
  • Genetic Mutations
  • Genomics
  • Goodpasture Syndrome
  • Hypoplastic Left Heart Syndrome
  • Immunesystem
  • Incidence
  • Information System
  • Joubert Syndrome
  • Kasabach-Merritt Syndrome
  • KAT6A Syndrome
  • Kikuchi Disease
  • Krabbe Leukodystrophy
  • Lesch-Nyhan Disease
  • Life -threatening Diseases
  • Lipoprotein Lipase Deficiency
  • Mackenzie Fox-Byrne
  • Marcus Gunn Jaw-winking Syndrome
  • Methemoglobinemia.
  • Mitochondrial Pathology
  • Monoclonal Antibody Products
  • Neglected Disease
  • Neurofibromatosis
  • Organic Academic
  • Orphan Drug Act
  • Orphan Indication
  • Orphan Indication
  • Orphan Medicinal Product
  • Orphan Pharmaceutical Product
  • Orphan Status.
  • Orphan Status.
  • Periodic Disease
  • Pharmacokinetics And Pharmacodynamics
  • Pharmacokinetics And Pharmacodynamics
  • Prevalence
  • Rare Bone Diseases
  • Rare Child Diseases
  • Rare Chromosome Disorders
  • Rare Congential Disorders
  • Rare Disability
  • Rare Diseases Awareness
  • Rare Genetic Diorders In Humans
  • Rare Medicinal Technology
  • Rare Pediatric Disease
  • Re-emerging Infectious Diseases
  • Schnitzler Syndrome.
  • Sepsis
  • Septicaemia
  • Sexually Transmitted Diseases
  • Sexually Transmitted Infections
  • Skin Diseases
  • Surgical Site Infections
  • Thrombocytopenia-Absent Radius Syndrome
  • Treatment For Infectious Diseases
  • Tuberculosis
  • Ultra Orphan Drug
  • Ultra Rare Disease
  • Unidentified
  • Uric Acid
  • Urinary Tract Infections
  • Vaccines And Vaccination
  • Very Rare Disease
  • Viral Infection
  • Zoonosis