6^th International Conference on Rare Diseases & Orphan Drug June 17-18, 2019 MENA Plaza Hotel Albarsha | Dubai, UAE

TRACK-1: Mystery Diagnosis of Rare Diseases

Determination and the treatment of a rare disease end up confounded due to the way that numerous medicinal services providers may have confined contribution with the identification of the particular disease condition with the diagnosis of rare diseases. Also, examination analysis before manifestation onset or diagnosis early stage in the disease can be challenging. Biopharmaceutical specialists in the field have used new advances and the creating exploratory appreciation of numerous rare diseases to create and analyse ground breaking therapies in the course of the most recent 10 years. In the most recent decade, in excess of 230 new orphan therapeutic medications were recognised by the U.S. Food and Drug Administration (FDA). In 2015 alone, about half (47%) of novel most recent medication confirmations were for rare diseases. Disease specific treatments should be created for beneficial outcomes in treatment of Rare Diseases.

TRACK-2: Rare Brain Disorder

Brain is body's control centre. It's a part of the nervous system, which likewise incorporates the spinal cord and a substantial system of nerves and neurons. Together, the nervous system controls everything from your senses to the muscles all through your body. When your brain is damaged, it can influence a wide range of things, including your memory, your sensation, and even your identity. Brain disorders incorporate any conditions or inabilities that influence your brain. Brain injuries are regularly caused by blunt trauma. Creutzfeldt-Jakob disease (CJD) is a rare, degenerative, fatal brain disease.  It influences around one individual in each one million every year worldwide in the United States there are around 350 cases per year.  CJD generally shows up in later life and runs a quick course. 

TRACK-3: Rare Oncology Diseases

Rare cancers can originate in any organ of the bodyincorporate gastrinomas, childhood acute myeloid leukaemia, schwannomas, retinoblastomas, or thymomas. For a portion of these diseases, specific medications have not yet been produced. All in all, in any case, inquire about in is greatly dynamic, with numerous medications either or being developed. The various kinds of incorporate tumours of the body and haematological of the blood. independently these malignancies are uncommon, as a gathering, they of individuals with cancer. Treatment for is typically collaboration, with general professionals, oncologists, haematologists, radiation specialists, and different with each other.

TRACK-4: Rare Genetic Diseases

The human ordination is continually exposed to varied sources of desoxyribonucleic acid injury. Rare genetic diseases are specifically caused by changes in genes or chromosomes, often go from one generation to next. Most of these diseases are regularly connected with severe or life threatening features. Numerous rare diseases seem early in life, and around 30% of children with rare diseases will die before achieving their fifth birthday. With a single analysed patient just, ribose-5-phosphate isomerase deficiency is viewed as the rarest known genetic disease. Frequency of a single rare genetic disease can fluctuate broadly yet is generally very infrequent, usually to or less than one per 1,00,000 births. A disease is viewed as rare if it influences less than 2,00,000 individuals in the United states, or less than five for every 10,000 individuals in the European Union.

TRACK-5: Rare Skin Diseases

Skin disorders differ significantly in manifestations and severity. They can be brief or changeless, and might be painless or painful. Some have situational causes, while others might be hereditary. Some skin conditions are minor, and others can be dangerous. Out of these, chronic skin conditions are available from birth, while others show up all of a suddenly later in life. The reason for these disorders isn't constantly known. Numerous permanent skin disorders have compelling treatments that empower broadened times of reduction. However, they're serious, and side effects can reappear at any time. Contact dermatitis is a standout amongst the most well-known word related illnesses.

TRACK-6: Rare Diseases in Anaesthesiology

Anaesthesia is getting to be more secure over the previous decades. This procedure was driven by the advancement of new analgesics and the foundation of present day safety ideas. Then again anaesthetists advantage from significant pathophysiological knowledge and the skill of making an interpretation of patient particular conditions into individual perioperative hazard. But in case of rare diseases if there should be an occurrence of it, this idea is frequently inadequate with regards to particular knowledge about the patient conditions and its significance for anaesthesia.

TRACK-7: Rare Pulmonary Diseases

Idiopathic pulmonary fibrosis (IPF) is a prolonged lung disorder considered by thickening, scarring (fibrosis) and stiffening of tissue within the lungs. Affected individuals develop shortness of breath and advanced lung disease. Ultimately, IPF results in life-threatening difficulties such as respiratory failure. Lymphangioleiomyomatosis (LAM) is a rare, advanced lung disease that mostly affects women. Interstitial lung diseases (ILD) are a set of rare lung conditions that effect chronic breathlessness. The problem typically develops over the age of 50 years, and can affect both men and women, and also children.              

TRACK-8: Rare Obstetrical Diseases

The most widely recognized rare condition that involves in obstetrical is Gestational trophoblastic disease (GTD), a rare group of diseases that develop in the uterus during an abnormal pregnancy. It arises when abnormal cells or tumours propagate in the tissues that are made following conception that would normally surround the embryo and develop into the placenta. The tumours are denoted to as "moles." Most GTDs are gentle, or noncancerous, however, some forms may improve into cancer and spread to other parts of the body. All methods of the disease can be treated and in most cases alleviated.

TRACK-9: Rare Oral Diseases

Oral-facial-advanced (OFDS) is an umbrella term for no less than 10 obviously unique hereditary disorders that are described by irregularities and flaws in the improvement of the structure of the oral cavity including the mouth, tongue, teeth, and jaw; the advancement of the facial structures including the head, eyes, and nose; and the fingers and toes; alongside varying degrees of mental disorder. The introduction of signs and side effects is to a great degree varied, making determination difficult. OFDS type I is the most widely recognized of these disorders, and it is very rare.

TRACK-10: Rare Haematological Diseases

At the point when the typical method of blood cell development and advancement separates, certain cell comprises can be delivered in abnormally large amounts or in abnormal form. This offer arises to blood diseases.  There are many conditions affecting the human haematological system including anaemia-type red blood cell conditions, white platelet dysfunctions related with different malignancies, immuno-disorders, platelet-based variations that influence coagulation, and plasma-based issues (e.g., haemophilia A and B, von Willebrand disease).

TRACK-11: Rare Aging Diseases

Aging is a combination of degenerative changes that happen in living beings that do not have the capacity to constantly recover. Age is a main risk factor for most collective neurodegenerative diseases.  The most widely recognized rare condition that involves in Aging is Progeria, an extremely rare autosomal dominant genetic disorder in which symptoms approaching characteristics of aging are revealed at a very early age. Progeria is one of several progeroid syndromes. Those born with progeria typically live to their teenager to early twenties. It is a genetic condition that happens as a new mutation, and is hardly inherited, as carriers generally do not live to reproduce. Studies have demonstrated that 37.5% had conventional life expectancy, 25.7% potentially lethal during childbirth or before 5 years of age, 36.8% reduced life expectancy.

TRACK-12: Rare Metabolic Diseases

The phrase metabolism related to all the chemical reactions taking place in the body to convert or use energy.  Catalysts assume a key part in a large number of these procedures and changes in their capacity, subsequently, of hereditary transformation can prompt issues in these pathways. Inherited metabolic disorders are genetic conditions that result in metabolism problems Metabolic changes can give incredible assorted qualities of signs and manifestations like episodic illness-anorexia, vomiting, and gastric metabolic disorder, lethargy, cardiomyopathy, muscular, gastrointestinal. Drug treatment is utilized to treat metabolic rare conditions; however this represents an oversees challenge to community and hospital pharmacists as they are not accessible in an appropriate formulation for administration.

TRACK-13: Rare Diseases of Genitourinary System

Numerous rare diseases affect the kidneys and the urinary system, some inadequate to the urinary system itself (e.g., membranous nephropathy), and some part of a condition (e.g., 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 (e.g., Goodpasture syndrome). Many rare types of vasculitis can also cause major kidney problems (e.g., Henoch-Schonlein purpura nephritis). The most widely recognized rare condition that involves the genitourinary System is Emphysematous cystitis (EC), a rare infection of the urinary tract that effects in gas production in the bladder. It is more common in diabetic and female patients, and can be associated with more serious complications, including pyelonephritis.

TRACK-14: Rare Musculoskeletal Diseases

Rare musculoskeletal diseases have diverse symptoms. Some cause muscle weakness or pain, sometimes affecting only specific muscle groups, while others cause skeletal deformation or weakness. In many conditions, other body systems are also affected. Often, symptoms can begin gradually but then worsen as muscles or bones become permanently damaged. The most widely recognized rare conditions that involve the musculoskeletal system are Duchene muscular dystrophy; arise from genetic problems in the muscles themselves, while others involve the nerves, motor neurons, or the bones. A correct diagnosis may come from a neurologist, rheumatologist, orthopaedist, or endocrinologist, numerous physicians, and allied health professionals can be of great service in managing a musculoskeletal disease.

TRACK-15: Rare Paediatric Diseases

The majority of rare diseases affect children, most of whom have a primary genetic source for their condition. However, making a molecular diagnosis with current technologies and knowledge is often still a challenge. Pediatric genomics is a rapidly developing field but it is not fine developed, that challenges this problem by integrating next-generation sequencing technologies, especially whole-exome sequencing and whole-genome sequencing, into research and medical workflows. This challenging multidisciplinary approach, shared with the rising accessibility of population genetic data variation, has already resulted in an increased discovery rate of connective genes and in a developed diagnosis of rare pediatric disease. Substantially, for affected individual family members, a good and systematic 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-16: Rare Diseases of Endocrine System

Despite the fact that various endocrine system disorders, for example, diabetes, thyroid, polygenic disorder, and osteoporosis are among the most extreme normal and understood therapeutic conditions, a few patients who have been referred to an endocrinologist will require treatment for a rare disease. A number of these rare diseases is risky and rapid myriad Sequelae and secondary diseases. Gland-based hormonal imbalances ensuing in progressive abnormalities and related difficulties are among these diseases, weight regulation issues and physical abnormalities and frequent oncogenic and non- oncogenic endocrine-related tumour disorders.  Rare endocrine diseases can hypothetically impact any organ system, on the other hand.

TRACK-17: Rare Cardiac Diseases

The most widely recognized inherent heart defect related to Kounis syndrome might be an episode of angina or even a heart attack activated by an allergic reaction. The syndrome was named later Professor Nicholas G Kounis, a Greek cardiologist, who initiate that the syndrome is caused by chemical substances, for example, histamine, which are discharged by the body during an allergic reaction. These chemicals compounds cause the arteries that supply the heart with blood to go into fit, in this manner constraining the bloodstream and causing angina-like chest pain. Torsade de pointe is a risky anomalous heart rhythm that can ready sudden cardiovascular death.

TRACK-18: Orphan Drugs- Development Trends and Strategies

An Orphan drug might be a medicative item produced for the treatment of a rare disease. Analysis and the treatment of a rare disease wind up complex due to the conditions that various healthcare providers may have confined contribution with the recognizable proof of the specific disease condition with the diagnosis of rare diseases. Biopharmaceutical specialists in the field have used new advances and the creating investigative appreciation of numerous rare diseases to create and analyze remarkable treatments in the course of the most recent 10 years. In 2015 alone, about half (47%) of novel latest drug approvals were for rare diseases. Numerous blockbuster medications will lose their eliteness in next 5 years. In this way, the current economic situation in addition to the tremendous generic competition moved the focal point of pharmaceutical organizations from the basic medicines to the new business model — niche busters, also called orphan drugs.

TRACK-19: Drug Approvals for Rare Diseases

The Orphan Drug gives orphan remaining to medication and biologics that area unit illustrated as those gathered for the safe and effective treatment, assignment or impedance of rare diseases that affect less than two hundred people inside the U.S., or that affect more than two hundred people anyway don't appear to be relied upon to recoup the costs of creating and promoting a treatment drug. The objective of the Orphan store Clinical Trials Grants Program is to support the clinical improvement of stock to be utilized in rare diseases or conditions. Last, the Pediatric Medical Device Safety and Improvement Act of 2007 (Public Law 110-85) licenses HDE approved devices expected to be utilized in pediatric patients or in an exceedingly pediatric populace and affirmed.

TRACK-20: Future Hereditary of Rare Diseases and Orphan Drugs

Work in the course of recent years has brought about the identification of qualities in charge of ~50% of the evaluated 7,000 rare monogenic infections and it is anticipated that the majority of the rest of the disease-causing qualities will be distinguished constantly 2020 and likely sooner. This checked acceleration is the aftereffect of dramatic improvements in DNA-sequencing innovations and the related examinations. We scrutinize the fast development of rare-disease hereditary analysis and effective techniques for quality identification. We feature the effect of discovering rare-disease-causing genes, from clinical diagnostics to bits of knowledge picked up into biological mechanisms and common diseases. Last, we investigate the expanding therapeutic opportunities and difficulties that the subsequent development of the ' Atlas ' of human hereditary pathology will bring.

Importance and Scope

Rare diseases are generally hereditary thus they chronic. It is measured that no less than 80% of them have recognized hereditary origins. Other rare diseases are the consequence of contaminations and hypersensitivities or it might be because of degenerative and proliferative causes and discovering the well-advanced medications in Rare Diseases and Orphan Drugs. There are in excess of 6000 rare diseases. Numerous rare diseases are analyzed at the time of adolescence, making diagnostic awareness and learning on treatment and tend to pediatricians.

Rare Diseases Meet 2019 is rising up with an energizing and high knowledge gaining conference program this year additionally which incorporates whole lectures, symposia, workshops on a collection of subjects, poster presentations and different programs for members from all through the world. We welcome you to go along with us at the Rare Diseases and Orphan Drug Conference 2019 in Dubai where you will make sure to have an important involvement with researchers from around the globe. The Rare Diseases Conference plans to broaden the achieve, effect and trade of insightful thoughts through innovative technology, determined commitment, and remarkable administration. It expects to provide global coordinated effort and trade of far-reaching and cutting-edge data on fundamental, translational, and linked clinical research on rare diseases and Orphan Drugs.

Why Dubai?                             

Dubai is a city in the United Arab Emirates, situated inside the emirate. The emirate of Dubai is situated on the southeast shoreline of the Persian Gulf and is one of the seven emirates that make up the nation. It has the most population in the UAE (2,106,177) and the second-biggest land an area by region (4,114 km2) after Abu Dhabi. It is one of only a handful couple of urban communities on the planet that has withstood such a quick transformation from an obliging start as a pearl-jumping focus - to one of the quickest developing urban areas on earth. Dubai nowadays is a business endeavour, exchange and supply centre. It is conjointly thought of dynamic core of the Arabian Gulf area. With year-round sunshine, fascinating deserts, lovely beaches, luxurious hotels and shopping malls, eye-catching heritage attractions and a prosperous business community, Dubai receives millions of leisure and business visitors each year from around the world is one of the few cities in the world that has withstood such a rapid conversion- from a courteous beginning as a pearl-diving centre - to one of the fastest growing cities on earth and has attained itself the name of being the ‘gateway between the east and therefore the west'.

·         Jumeirah Mosque

·         Miracle Garden

·         Hatta Rock Pools

·         Dubai Creek Golf & Yacht Club

·         Emirates Towers

·         Palm Jumeirah

·         Dubai Museum

·         Atlantis Palm Jumeirah

·         Dubai Aquarium and Underwater Zoo

·         Burj Khalifa

·         Dubai Marina Walk

Conference Highlights

·         Mystery Diagnosis of Rare Diseases

·         Rare Brain Disorders

·         Rare Oncology Diseases

·         Rare Genetic Diseases

·         Rare Skin Diseases

·         Rare Diseases in Anaesthesiology

·         Rare Pulmonary Diseases

·         Rare Obstetrical Diseases

·         Rare Oral Diseases

·         Rare Haematological Diseases

·         Rare Aging Diseases

·         Rare Metabolic Diseases

·         Rare Diseases of Genitourinary System

·         Rare Musculoskeletal Diseases

·         Rare Paediatric Diseases

·         Rare Diseases of Endocrine System

·         Rare Cardiac Diseases

·         Orphan Drugs- Development Trends and Strategies

·         Drug Approvals for Rare Diseases

·         Future Hereditary of Rare Diseases and Orphan Drugs

Top Universities across Global :

·         Harvard University

·         University of Oxford

·         University of Cambridge

·         Johns Hopkins University

·         Stanford University

·         Yale University

·         University of California, Los Angeles (UCLA)

·         Imperial College London

·         University of Melbourne

·         Karolinska Institute

·         American University of Sharjah

·         University of Sharjah

·         Masdar Institute of Science and Technology

·         Abu Dhabi University

·         Dubai Pharmacy College                            

Top Universities in UAE:

·         Gulf Medical University

·         Ras al-Khaimah Medical and Health Sciences University

·         Dubai Medical College

·         College Of Medicine and Health Sciences

·         United Arab Emirates University

·         American University of Sharjah

·         University of Sharjah

·         Masdar Institute of Science and Technology

·         Abu Dhabi University

·         Dubai Pharmacy College

Top Universities in Dubai :

·         Biotechnology university college dubai

·         American University in Dubai

·         Dubai Medical College

·         Dubai Pharmacy College

·         Ras al-Khaimah Medical and Health Sciences University

·         Mohammed Bin Rashid University Of Medicine and Health Sciences

·         mohamedsyed medical university dubai

·         University City of Sharjah

·         British University in Dubai

·         Hamdan Bin Mohammed Smart University

Top Hospitals across Global:

·         Nuffield Health Bristol Hospital – The Chesterfield – Bristol, U.K.

·         Upper River Valley Hospital – New Brunswick, Canada

·         Hackensack University Medical Centre – Hackensack, New Jersey

·         Legacy Salmon Creek Medical Centre – Portland, Washington

·         Wooridul Spine Hospital – Seoul, South Korea

·         St. James’ Institute of Oncology/Leeds Cancer Centre – West Yorkshire, U.K.

·         Houston Methodist Hospital – Houston, Texas

·         Hartford Hospital – Hartford, Connecticut

·         Vale Hospital – Hensol, South Wales, U.K.

·         University of Pittsburgh Medical Centre – Pittsburgh, Pennsylvania

·         University of Texas MD Anderson Cancer Centre – Houston, Texas

·         UCLA Health: Ronald Reagan UCLA Medical Centre – Los Angeles, California

·         Mercy San Juan Medical Centre – Carmichael, California

·         Massachusetts General Hospital – Boston, Massachusetts

·         Ramkhamhaengs Hospital – Bangkok, Thailand

Top Hospitals in UAE:

·         Zulekha Hospital Sharjah

·         Emirates Hospital

·         Dubai Hospital (Govt)

·         Rashid Hospital (Govt)

·         Medcare Hospital

·         Canadian Specialist Hospital

·         Cosmolaser Medical Center

·         Al Zahra Hospital Sharjah

·         University Hospital Sharjah

·         Al Amanah Medical Center

·         Sunny Medical Centre LLC, Shahba

·         New Sunny Medical Centre LLC, Rolla

·         Al Salam Medical Center

·         Dr Fatima Nooh Medical Center LLC

·         Aster Medical Centre – Sharjah

·         Marina Medical Centre – Sharjah

Top Hospitals in Dubai:

·         Alkhayal Medical Centre

·         Universal Hospital - Medical Center

·         Mediclinic Arabian Ranches

·         Al Zahra Medical Centre

·         German Heart Centre Bremen FZ-LLC

·         Medeor Downtown Medical Centre

·         Zulekha Hospital Dubai

·         Conceive Gynaecology and Fertility Hospital JLT

·         Magnum Gulf Medical Center Silicon Oasis DSO

·         Genesis perinatal care clinic

Top Pharmaceutical companies in Global:

·         Novartis

·         Mylan Laboratories Limited

·         Hetero Labs Limited Unit 3

·         Natco Pharma Limited

·         Suven Pharmaceuticals Ltd

·         S M S Pharmaceuticals Ltd.

·         Boehringer Ingelheim

·         Al Hayat Pharmaceuticals Company

·         Medpharma

·         Life Pharma FZE

·         Gulf Pharmaceutical Industries 

Top Pharmaceutical companies in UAE:

·         Global Pharma

·         Pharmatrade L.L.C

·         Boehringer Ingelheim

·         Al Hayat Pharmaceuticals Company

·         Medpharma

·         Life Pharma FZE

·         Gulf Pharmaceutical Industries 

·         Mylan

·         Clinart International

·         Pharmaworld LLC

Top Pharmaceutical companies in Dubai :

·         Boehringer Ingelheim

·         MPC Healthcare

·         Bayer Middle East FZE

·         Gilead Sciences Middle East Office

·         Novo Nordisk Pharma Gulf Fz Llc

·         Hoffman La Roche Dubai Branch

·         Pharma House Pharmaceuticals LLC

·         NewBridge  Pharmaceuticals Scientific Office

·         Gulf Drug LLC

·         Bristol-Myers Squibb FZ-LLC

Industries Associated with Rare Diseases Research Worldwide

·         AbbVie

·         Actelion

·         AFM-Telethon

·         Alexion

·         Amgen

·         Amicus Therapeutics

·         AstraZeneca

·         Baxter

·         Bayer

·         Biogen

·         BioMarin

·         Bioxcel

·         Bristol-Myers Squibb

·         Celgene

·         Cydan

·         Eli Lilly

·         Genentech

·         Gilead Sciences

·         GlaxoSmithKline

RELATED CONFERENCES

• 14th International Conference on Infectious Diseases, Prevention and Control,  March 21-22, 2019 Dubai, UAE

• 2nd Annual Congress on Bacterial, Viral and Infectious Diseases , June 17-18, 2019 Dubai, UAE

• 6th International Congress on Infectious Diseases , Feb 25-26, 2019, London, U.K

• 12th World Congress on Virology and Infectious Diseases ,  March 13-14, 2019, Singapore

• 9th World Congress on Rare Diseases and Orphan Drugs,  June 17-18, 2019 Berlin, Germany

• Annual Meeting on Infectious Diseases , July 29-30, 2019 Amsterdam, Netherland

• 10th Annual Congress on Rare Diseases and Orphan Drugs, September 23-24, 2019 Toronto, Canada

• 15th World Congress on Infection Prevention and Control,  October 21-22, 2019 Zurich,  Switzerland

• 5th EORTC Quality of Life and Cancer Clinical Trials Conference, May 16-17, 2019, Brussels, Belgium

• American Society of Clinical Oncology (ASCO) 2019 Annual Meeting, May 31- June 4, 2019, Chicago

• Rare Disease Innovation and Partnering Summit, June 12-13,2019

• 6^th International Conference on Brain Disorders and therapeutics, July 1-2, 2019, Paris, France

• 14^th Congress of European Association of Neuro-Oncology (EANO), September 19-22,2019, Lyon, France

   

Europe:

European Society of Cardiology; Brain Research Society of Finland; Israel Society of Anaesthesiologists; European Academy of Paediatrics; European Society of Pathology; Nordic Pediatric Society; European Society for Clinical Virology; European Society of Clinical Microbiology and Infectious Diseases; Federation of European Microbiological Societies; European federation of parasitologists; Irish Society for Parasitology; Irish Cardiac Society; German Cardiac Society; Romanian Association of Hypertension; Netherlands Society of Cardiology

USA and Canada:

Heart Failure Society of American; American Society of Echocardiography; Society of Invasive Cardiovascular Professionals; American Neurological Association; Canadian Neurological Sciences Federation; American Society for Clinical Pathology; Society for Industrial Microbiology and Biotechnology; Canadian Society of Microbiologists; Canadian Pediatric Anaesthesia Society; American Society of Anaesthesiologists; American Academy of Ophthalmology; Canadian Ophthalmological Society

Asia Pacific:

Australian Society of Ophthalmologists; Australian Cardiovascular Health and Rehabilitation Association; Indonesian Heart Association; Indonesian Society of Anaesthesiologists and Intensive Therapy; Australian Society of Anaesthetists; Korean Society of Anaesthesiologists; Singapore Society of Anaesthesiologists; Singapore Society for Microbiology and Biotechnology; Australasian Virology Society; Australian Society for Parasitology; Dental Hygienists Association of Australia

Middle East:

Iranian Heart Association ; Oman Heart Association; World Federation Of Societies of Anaesthesiologists; Egyptian Society of Neurology, Psychiatry and Neurosurgery; Egypt Association for Research in Vision and Ophthalmology; Egyptian Society for Microbiology and Immunology; Egyptian Society of Virology; Sudanese Association of Pathologists ; Syrian Pharmacists Association; Oral History Association of South Africas