10^th International Conference on Rare Diseases & Orphan Drug December 23-24, 2023 Dubai, UAE

An orphan disease is a rare disease (according to US criteria, a disease that affects fewer disregards (such as cholera, typhoid, tuberculosis, and malaria) because it is far more prevalent in developing countries than in the developed world. Public health practitioners use a combination of disciplines that include basic science, epidemiology, behavioural research, clinical research, health care services, statistics, economics, and policy to identify the primary or secondary causes of health threats and then consistently prevent or decrease these causes in entire populations.

• Cystic fibrosis
• Lou Gehrig's disease
• Tourette's syndrome

An orphan drug can be determined as one that is used to handle an orphan disease. For example, haem arginate, used to control small cintermittent porphyria, variegate porphyria, and hereditary coproporphyria is an orphan drug. A medicinal product which is came into the source as orphan drug is one that has been developed especially to treat a rare medical condition which is found, the condition itself being referred to as “orphan disease.” It may be defined as drugs that are not evolved by the pharmaceutical industry for economic reasons but which respond to public.

• Acute intermittent porphyria
• Variegate porphyria

Neglected tropical diseases (NTDs), such as lymphatic filariasis, dengue, leishmaniasis, and trachoma are called "neglected," because they generally afflict the world's poor and historically have not received as much awareness as other diseases. They are group of tropical diseases which are mostly seen in low income population and low developing countries like Africa, Asia and America. In this some are relatively expensive and some are not curable diseases.  The deaths with tropical diseases are recently come into the analysis and it seems to be higher in low developing regions.

• Buruli Ulcer.
• Chagas Disease.
• Cysticercosis.
• Dengue Fever.

Valinemia is a very   infrequent metabolic disorder. It is distinguish by elevated stages of the amino acid valine in the blood and urine caused by a deficiency of the enzyme valine transaminase. This enzyme is requiring in the breakdown (metabolism) of valine. Obesity resembles some major health risks it can lead to impair of quality of life include the most frequent type2 diabetes cardio vascular diseases and certain type of cancers .Diabetes is most commonly seen metabolic disorders which is acquired by genetic factor.

•    Familial hypercholesterolemia.
•   Gauche disease.
•   Maple syrup urine disease.
•   Mitochondrial encephalopathy, lactic acidosis, stroke-like episodes (MELAS)

Allergies, also known as allergic diseases, are a number of Pathology caused by hypersensitivity of the immune system to typical yon toxic material in the environment. These diseases include food allergies, dermatitis, hay fever, atopic dermatitis, allergic asthma, and anaphylaxis Allergic diseases and the major symptoms occur because of an growing immune system that reacts to things that are usually harmless, such as pollens, pet dander etc. They can be easily diagnosed if we are under good control of it.

• Allergy Pathogenesis
• Asthma
• Complement-Related Abnormalities
• Immunodeficiency

The biopharmaceutical investigator has made immense progress in move along with innovation for rare diseases, some of the rare diseases unmoving do not have some therapy options, act as a remarkable unmet need for patients. We've seen unbelievable proceed in the growth of medicines to treat patients with rare diseases as experimenters reveal the molecular and genomic drivers of many orders. The fatal and genetic conditions in the children was striking features resembles premature aging.

• Water allergy
• Foreign accent syndrome
• Laughing Death

The medicinal significant delegate as an orphan drug is one that has been growing specifically to treat a rare medical disorder, the condition describes as “orphan disease and also explain as drugs that are not made of implementing by the pharmaceutical industry for economic purposes. Which acts as public offer reason for assumption to families and individuals to bare from rare diseases like Batten Disease, Spinal Muscular Atrophy, and Duchenne? The main reason behind orphan drug is that cure any other future orphan rare diseases even any cause of deficiency.

• RPI Deficiency
• Hutchinson-Gilford Progeria

The productive speak to rare disease therapies have loose short of providing desperately require interventions for the expansive majority of rare disease patients. That particular the patients' and doctors' struggling in determine a diagnosis  frequently due to negligent symptoms, masquerading syndromes, the scarcity of the disease or condition or the patient's case being an atypical manifestation of condition or disease. As it is named by the program partner in organizing rare diseases.

•  Epilepsy
• Myasthenia gravis
• Alpha 1-antitrypsin deficiency
• Heart disease

Numerous suspected cases of genetic disorders endure undiagnosed or misdiagnosed due to the absence of connected clinical as well as diagnostic assets in the region, bring about patients to face an immense psycho-socio-economic emergency and numerous a time suffer long-lasting with their ailment. The disorganization that affects one child can be very dissimilar from how it affects another. They are different cause of rare diseases mainly the genetic growth directly caused by chromosomes in some cases it may also cause from one generation to another generation.

• Polymelia
• Neurofibromatosis
• Diprosopus

In children, difficulties of many urinary systems incorporate acute and chronic kidney non-success, urinary tract infections, block along the urinary tract, and abnormalities in attendance at birth. Diseases of the kidneys frequently make temporary or permanent changes to the small functional formation and vessels interior the kidney. Intravenous Urography This test requires taking an X-ray of the urinary tract. Before X-rays are removed, a pigment is injected to make urine obvious on the X-ray, which can show any blockages in the urinary tract or issues with the kidneys.

• Acute Kidney Injury
• Alport Syndrome
• Nephropathic Cystinosis

Sexually dispatched diseases (STDs), or sexually transmitted infections (STIs), are infections that are passed from one person to another through sexual contact. The contact usually happens through sex. But sometimes they can spread through another intimate physical contact. This is because of some STDs, like herpes and HPV, are spread by skin-to-skin contact which automatically happens the body. Human virus can easily spread by the sexual contacts such as

• Papillomaviruses
• Hepatitis B virus
• Human Immunodeficiency Virus (HIV)

To examine the association of rare genetic diseases influences skeletal development, socio-demographic characteristics, and oral health-related behaviours with dent clinical measures in children and adolescents. Rare diseases of genetic origin are often related with other co-morbid conditions, which are often characterized by debilitating and chronically degenerative conditions associates to phenotypes2. Rare Oral diseases can occur in any form like cancer, immunity deficiency, and metabolic conditions.

• Gigantism
• Maple syrup urine disease
• Ochoa syndrome
• Foreign accent syndrome 
• Carcinoid syndrome

Drugs that are evolved specifically to treat rare diseases – called “orphan drugs” – often remain economically of Rare Disease Day, it’s worth reanalyse a series of Healthcare Triage videos we updated last year that majorly focuses on orphan drugs, what’s wrong with the undergoing system, and how we might get ridge out of it. Can we people come out of this undergoing worst system situation? An orphan drug is usually defined as that one used to treat orphan diseases for example heam arginate and many more. 

• Acute intermittent porphyria
• Variegate porphyria
• Hereditary coproporphyria
• Haem arginate

Fungal diseases can affect easily as it keeps on spreading the infection in the body will grow faster Mild fungal skin diseases can look like a rash and are very common. Fungal diseases in the lungs are most similar to other illnesses such as bacterial or viral pneumonia which might cause fever symptoms. Some fungal diseases like fungal bloodstream and mucormycosis infections can be deadly. The major cause of fungal diseases is fingernails as we don't usually wash our hands the infection directly goes into the mouth.

• Aspergillosis
• Blastomycosis
• Candidiasis

A rare disease is defined as a disease or disorder that affects fewer than estimated at any given time. Among these, according to the National Institutes of Health (NIH), are more distinct rare diseases that usually affect the endocrine system. Their symptoms may seem rare in the body as vague, which can make it exacting to determine exactly what is wrong. Such “medical mysteries” often leave physicians to confound and patients going visiting doctor after doctor, searching for answers with no clear diagnosis.so, people randomly losing their lives.

• Ribose-5 phosphate isomerase
• Down Syndrome
• Thalassemia

As youngsters with a genetic syndrome transition to adult medical care, they may be mentioned to obstetrician-gynaecologists for routine preventive or contraceptive services, screening, or counselling for sexually circulated infection, or for menstrual management. Although some genetic syndromes have no physical impairment, others have similar ones; therefore, education and gynaecologic care should be based on a patient’s psychological and physical capabilities. It is important to mention that adolescents with or without a genetic syndrome are sexual beings.

• Acute Abdomen and Pregnancy
• Adrenal Disease and Pregnancy

Clinical research is central to the National Institutes of Health (NIH) goals of improving health, increasing health, and reducing the burden of illness and disability. For example, through clinical research we can gain some insights and answers about the safety and efficacy of drugs and other therapies which can aware publish with some researches. To encourage patient patience and commitment, to encourage trial participants to actively share their online experiences through social media, patient forums, or orally.

• Leveraging patient
• Volunteer advocacy

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. Rare diseases or orphan diseases are conditions that affect a small percentage of population at any given time. According to the U.S. Food & Drug Administration (FDA), an orphan disease is defined as a disease or condition affecting less than 200,000 people in the country. Orphan Drugs Act under the U.S. legislation allows manufacturer to request FDA to grant an orphan drug status to drugs intended to treat a rare disease or condition.

The Objective of the "Global Rare Disease Drugs Market" report is to depict the trends and upcoming for the Rare Disease Drugs industry over the forecast years. Rare Disease Drugs Market report data has been gathered from industry specialist/experts. Although the market size of the market is studied and predicted from 2019 to 2028 mulling over 2016 as the base year of the market study. Attentiveness for the market has increased in recent decades due to development and improvement in the innovation

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.

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 Diseases. 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. According to Statistics of MRC, the Global Orphan Drug market is estimated at $145.89 million in 2018 and is expected to reach $265.63 million by 2022 growing at a CAGR of 10.5% from 2018 to 2025.

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'.

• 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
• Every Life 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 Centre 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 Centre for Orphan Disease Research
• U.S. Food and Drug Administration
• UCLA Health
• US hospital for Rare Disease 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
• North-western 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

• 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 Research, 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