Rare Diseases 2020


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

  • Study worldwide patterns in uncommon infections and its advances in the remedial and demonstrative market.
  • Talking about key win-win coordinated efforts to help quicken uncommon infection clinical medication advancement, and improve connections between sedate engineers and patients to consider the two gatherings' inclinations.
  • Hear progressively about how quiet commitment by coordinating the patient point of view into the medication advancement process through patient promotion, tolerant driven research, and patient gatherings.
  • Learn about how the regulatory landscape for drug approvals is different between countries, and why it is important to keep informed about the regulations and guidelines of each region.
  • Increase information on various uncommon sicknesses and their novel difficulties, just as how treatment techniques can be moved to other Rare Diseases.
  • Introducing cell and gene therapies to rare disease treatment - learn how gene therapy methods can improve the treatment of rare diseases and why it is becoming more commercially successful. 



A rare/orphan disease is outlined as a condition that affects fewer than 200,000 people. This definition was made by Congress inside the Orphan Drug Act of 1983. There may be as many as 7,000 rare diseases and 70% of them have no form of treatment. Regulatory edges like longer market exclusivity, breakthrough designations, reduced fees, and tax incentives are all encouraging investment. However, the promoting procedure and lifecycle for a rare disease drug are altogether different from a mass-market item and require specific aptitudes and information. Rare Diseases Congress 2020 will be the best stage for every one of the specialists, analysts, eminent Scientists, examine researchers, understudies who are working in this field over the globe under a solitary rooftop to trade their insight identified with Rare Diseases and Orphan Drugs. This international event is an effort to find an alternative for invasive imaging technique against rare diseases like Zellweger syndrome, Muscular Dystrophy, Alkaptonuria, Angelman syndrome, Prader-Willi syndrome, Tay Sachs disease, Fragile X syndrome, Gaucher disease as well as many others.

Conference Highlights

  • Rare Infectious Diseases and Immune Deficiencies
  • Different types of Rare Diseases
  • Mystery Diagnosis of Rare Diseases
  • Challenges in Rare Diseases Treatment
  • Rare Diseases in Cancer
  • Orphan Drugs- development trends and strategies
  • Clinical Research on Orphan Drugs
  • Entrepreneurs Investment Meet


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

  • Directors, Board Members, Presidents, Vice Presidents, Deans and Head of the Departments
  • Researchers, Scientists, Faculties, Students
  • Rare Diseases Associations and Societies
  • Medical Colleges
  • Industrial delegates from Academia and Research
  • Industrial professionals from healthcare sectors biomedical companies
  • Medical Devices Manufacturing Companies
  • Drug Manufacturing Companies and Industries
  • Laboratory Technicians and Diagnostic Companies
  • Business Entrepreneurs and Industrialists

A Unique Opportunity for Advertisers, exhibitors, and Sponsors at Global Congress on Rare Diseases and Orphan Drugs (Rare Diseases 2020)


GBI Research's examination shows that the complete worldwide vagrant illness therapeutics showcase is relied upon to increment at a noteworthy compound yearly development pace of 13.1% from $2.3 billion in 2010 to $6 billion in 2019 in the US, the top five countries of Japan and Europe.

The ongoing Orphan Drug Report from Evaluate, which propelled as of late at BIO worldwide Convention, reveals insight into the market elements of vagrant medications — pharmaceutical items focused on uncommon illnesses, anticipating that deals will encounter a compound yearly the development pace of 7.4 percent between 2012 and 2019, nearly double that of the prescription drug market, excluding generics.  The report based on Evaluate Pharma data found that the worldwide orphan drug market is set to reach approximately $127 billion by 2019, doubling that of the overall prescription drug market

The worldwide Orphan Drugs showcase came to $84.9 billion every 2009 developing from $58.7 billion out of 2006 from $54.5 billion in 2005. The market is required to develop at a compound yearly development rate (CAGR) of almost 6% to reach $112.1 billion by 2014. The U.S. represented 51% of the market in 2009 and is required to develop at a CAGR of 8.9% to reach $65.9 billion by 2014. By 2020, vagrant medications will claim 19% of the absolute portion of physician recommended sedate deal barring generics, arriving at an astounding $176 billion in yearly deals, as per Andreas Hadjivasiliou, an expert with Evaluatepharma.


List of Rare Diseases

  •   Acrocephalosyndactylia
  •   Addison Disease
  •   Adie Syndrome
  •   Alagille Syndrome
  •   Amylose
  •   Amyotrophic Lateral Sclerosis
  •   Angelman Syndrome
  •   Angiolymphoid Hyperplasia with    Eosinophilia
  •   Arnold-Chiari Malformation
  •   Arthritis, Juvenile Rheumatoid
  •   Asperger Syndrome
  •   Bardet-Biedl Syndrome
  •   Barrett Esophagus
  •   Beckwith-Wiedemann Syndrome
  •   Behcet Syndrome
  •   Bloom Syndrome
  •   Bowen's Disease
  •   Brachial Plexus Neuropathies
  •   Brown-Sequard Syndrome
  •   Budd-Chiari Syndrome
  •   Burkitt Lymphoma
  •   Carcinoma 256, Walker
  •   Caroli Disease
  •   Charcot-Marie-Tooth Disease
  •   Chediak-Higashi Syndrome
  •   Chiari-Frommel Syndrome
  •   Chondrodysplasia Punctata
  •   Colonic Pseudo-Obstruction
  •   Colorectal Neoplasms, Hereditary Nonpolyposis
  •   Craniofacial Dysostosis
  •   Creutzfeldt-Jakob Syndrome
  •   Crohn Disease
  •   Cushing Syndrome
  •   Cystic Fibrosis
  •   Dandy-Walker Syndrome
  •   De Lange Syndrome
  •   Dementia, Vascular
  •   Dermatitis Herpetiformis
  •   DiGeorge Syndrome
  •   Diffuse Cerebral Sclerosis of Schilder
  •   Duane Retraction Syndrome
  •   Dupuytren Contracture
  •   Ebstein Anomaly
  •   Eisenmenger Complex
  •   Ellis-Van Creveld Syndrome
  •   Encephalitis
  •   Enchondromatosis
  •   Epidermal Necrolysis, Toxic
  •   Facial Hemiatrophy
  •   Factor XII Deficiency
  •   Fanconi Anemia
  •   Felty's Syndrome
  •   Fibrous Dysplasia, Polyostotic
  •   Fox-Fordyce Disease
  •   Friedreich Ataxia
  •   Fusobacterium
  •   Gardner Syndrome
  •   Gaucher Disease
  •   Gerstmann Syndrome
  •   Giant Lymph Node Hyperplasia
  •   Glycogen Storage Disease Type I
  •   Glycogen Storage Disease Type II
  •   Glycogen Storage Disease Type IV
  •   Glycogen Storage Disease Type V
  •   Glycogen Storage Disease Type VII
  •   Goldenhar Syndrome
  •   Guillain-Barre Syndrome
  •   Hallermann's Syndrome
  •   Hamartoma Syndrome, Multiple
  •   Hartnup Disease
  •   Hepatolenticular Degeneration
  •   Hepatolenticular Degeneration
  •   Hereditary Sensory and Motor Neuropathy
  •   Hirschsprung Disease
  •   Histiocytic Necrotizing Lymphadenitis
  •   Histiocytosis, Langerhans-Cell
  •   Hodgkin Disease
  •   Horner Syndrome
  •   Huntington Disease
  •   Hyperaldosteronism
  •   Hyperhidrosis
  •   Hyperostosis, Diffuse Idiopathic Skeletal
  •   Hypopituitarism
  •   Inappropriate ADH Syndrome
  •   Intestinal Polyps
  •   Isaacs Syndrome
  •   Kartagener Syndrome
  •   Kearns-Sayre Syndrome
  •   Klippel-Feil Syndrome
  •   Klippel-Trenaunay-Weber Syndrome
  •   Kluver-Bucy Syndrome
  •   Korsakoff Syndrome
  •   Lafora Disease
  •   Lambert-Eaton Myasthenic Syndrome
  •   Landau-Kleffner Syndrome
  •   Langer-Giedion Syndrome
  •   Leigh Disease
  •   Lesch-Nyhan Syndrome
  •   Leukodystrophy, Globoid Cell
  •   Li-Fraumeni Syndrome
  •   Long QT Syndrome
  •   Machado-Joseph Disease
  •   Mallory-Weiss Syndrome
  •   Marek Disease
  •   Marfan Syndrome
  •   Meckel Diverticulum
  •   Meige Syndrome
  •   Melkersson-Rosenthal Syndrome
  •   Meniere Disease
  •   Mikulicz' Disease
  •   Miller Fisher Syndrome
  •   Mobius Syndrome
  •   Moyamoya Disease
  •   Mucocutaneous Lymph Node Syndrome
  •   Mucopolysaccharidosis I
  •   Mucopolysaccharidosis II
  •   Mucopolysaccharidosis III
  •   Mucopolysaccharidosis IV
  •   Mucopolysaccharidosis VI
  •   Multiple Endocrine Neoplasia Type 1
  •   Munchausen Syndrome by Proxy
  •   Muscular Atrophy, Spinal
  •   Narcolepsy
  •   Neuroaxonal Dystrophies
  •   Neuromyelitis Optica
  •   Neuronal Ceroid-Lipofuscinoses
  •   Niemann-Pick Diseases
  •   Noonan Syndrome
  •   Optic Atrophies, Hereditary
  •   Osteitis Deformans
  •   Osteochondritis
  •   Osteochondrodysplasias
  •   Osteolysis, Essential
  •   Paget Disease Extramammary
  •   Paget's Disease, Mammary
  •   Panniculitis, Nodular Nonsuppurative
  •   Papillon-Lefevre Disease
  •   Paralysis
  •   Pelizaeus-Merzbacher Disease
  •   Pemphigus, Benign Familial
  •   Penile Induration
  •   Pericarditis, Constrictive
  •   Peroxisomal Disorders
  •   Peutz-Jeghers Syndrome
  •   Pick Disease of the Brain
  •   Pierre Robin Syndrome
  •   Pigmentation Disorders
  •   Pityriasis Lichenoides
  •   Polycystic Ovary Syndrome
  •   Polyendocrinopathies, Autoimmune
  •   Prader-Willi Syndrome
  •   Pupil Disorde
  •   Rett Syndrome
  •   Reye Syndrome
  •   Rubinstein-Taybi Syndrome
  •   Sandhoff Disease
  •   Sarcoma, Ewing's
  •   Schnitzler Syndrome
  •   Sjogren's Syndrome
  •   Sjogren-Larsson Syndrome
  •   Smith-Lemli-Opitz Syndrome
  •   Spinal Muscular Atrophies of Childhood
  •   Sturge-Weber Syndrome
  •   Sweating, Gustatory
  •   Takayasu Arteritis
  •   Tangier Disease
  •   Tay-Sachs Disease
  •   Thromboangiitis Obliterans
  •   Thyroiditis, Autoimmune
  •   Tietze's Syndrome
  •   Togaviridae Infections
  •   Tolosa-Hunt Syndrome
  •   Tourette Syndrome
  •   Uveomeningoencephalitic Syndrome
  •   Waardenburg's Syndrome
  •   Wegener Granulomatosis
  •   Weil Disease
  •   Werner Syndrome
  •   Williams Syndrome
  •   Wilms Tumor
  •   Wolff-Parkinson-White Syndrome
  •   Wolfram Syndrome
  •   Wolman Disease
  •   Zellweger Syndrome
  •   Zollinger-Ellison Syndrome