Track 1: Different Types of Rare Diseases
A rare disease is defined as a condition that affects less than 200,000 people. There are more than7, 000 rare diseases. Only few categories of rare diseases are tracked when a person is diagnosed. These include certain types of infectious diseases, birth defects, and cancers. It also comprises the diseases on newborn screening tests. Most rare diseases are genetic, and thus are present throughout the person's intact life, even if symptoms do not immediately appear.
-
Addison's Disease
-
Amyloidosis
-
Hereditary Angioedema
-
HoFH
-
Hyperkalemia
-
Hypophosphatemia
-
Kawasaki Disease.
Track 2: Rare Diseases and Their Causes
Rare diseases are a multiple heterogeneous group of diseases with a little in like manner aside from of their irregularity affecting with influencing the individuals. It is a health condition of low prevalence that affects a very less number of people compared with other diseases in the general population. Most of these diseases are caused by genital variations and hereditary inceptions. There are more than 7000 rare diseases.
-
single-gene mutations
-
cystic fibrosis
-
muscular dystrophies
-
Huntington disease
-
PI deficiency.
-
Fields Condition
-
Kuru
-
Methemoglobinemia
-
Hutchinson-Gilford proger
Track 3: Pediatric Infectious Diseases
Pediatrics is the branch of medicine science that deals with the medical care of infants, children, and adolescents. Infectious disease, are also known as infect ology, is a medical specialty that dealing with diagnosis, control and treatment of infections. An infectious disease is practice may consist of managing nosocomial infections, or it may be out-patient base. Pediatric genomics is a rapidly developing field but it is not well developed, that challenges this problem by integrating next-generation sequencing technologies, especially whole-exam 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.
Track 4: Rare Genetic Disorders
A disease or disorder is defined as rare in the Europe when it affects fewer than 200,000 people.at any given time. A rare disease is a condition that affects a small percentage of the population. ... With only three diagnosed patients in 27 years, ribose-5-phosphate isomerize deficiency is considered the rarest known genetic disease
Common Genetic Disorders: Down syndrome. Typically, the nucleus of an individual cell contains 23 pairs of chromosomes, but Down syndrome occurs when the 21st chromosome is copied an extra time in all or some cells.
-
Thalassemia
-
Cystic Fibrosis
-
Tay-Sachs disease
-
Sickle Cell Anemia
-
Learn More
-
Recommended
-
Sources
Track 5: Orphan Drugs, Their Potencies and Current Researches
An orphan drug is a pharmaceutical agent developed to treat the medical conditions, because they are so rare drugs, which would not be profitable to produce without government assistance. This condition is referred to as orphan diseases. “Orphan drugs" are medicinal products that intended to diagnosis, prevention and treatment of life-threatening serious diseases or disorders that are rare.
-
haemalginate
-
acute intermittent porphyria
-
variegate variegate
-
hereditary coproporphyria,
Track 6: Rare Aging and Endocrine Disease
Aging is a combination of degenerative changes that happen in living beings that doesn’t have the capacity to constantly recover. Aging is a main risk factor for most collective neurodegenerative diseases. The rare aging diseases are most common in children. The most common type of aging diseases is Progeria. The children turn older in at a very tender age. The life span of these children is not more than 13. The cause of this disease is due to sporadic mutation of the LMNA gene coding for a structural protein, Lamina A. Progerin is the truncated version of this structural protein. The diagnosis has been discovered but the cure has not yet been discovered for this disease. There are many life threatening aging diseases which are as follows:
• Premature aging syndromes
• Mysterious conditions
• Kallman syndrome
• Reactive attachment disorder
• Terminal leukodystrophy
Endocrine diseases in rare condition are caused due to hormonal changes, either increase or decrease in the release of hormonal secretions into the blood stream. Many diseases are enclosed in this particular group. They are: Addison’s disease, Cushing syndrome, Acromegaly, Diabetes insipid us, adrenocortical carcinoma, Anorchia, Autoimmune polyglandular syndrome, Gigantism and many more. These diseases are diagnosed and the cause is known but only some of them can be treated.
Track 7: Gene Therapy for Rare Diseases
Gene Therapy mainly involves in the alteration of genetic material within a cell or organism with an intention of curing of the diseases. 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 proof 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 anemia, hemophilia, 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.
-
Gene therapy for malignant melanoma
-
Gene therapy for malignant melanoma
-
Gene therapy for sickle-cell disease
-
Development of regenerative treatment models
-
Different vectors for gene therapy
-
Gene therapy product
Track 8: Rare Neurological Disorders
The Neurological disorders can be classified into two categories, those that affect the central nervous system are brain and spinal cord and also that affects the peripheral nervous system are few rare specified neurological disorders. This may occur in the infant during childbirth because of birth injuries and Structural disorders, such as brain or spinal cord injury, Bell's palsy, cervical spondylitis; carpal tunnel is inherited in an autosomal recessive manner. It is necessary to study the physiological defects of brain and muscle structures in LS for diagnosis and treatment.
-
Neuromuscular Disorders
-
Peripheral Neuropathy
-
Sciatic Pain
-
Neuron-immune Interaction
-
Neurovirology
-
Aicardi-Goutieres
-
Reflex Sympathetic Dystrophy
-
Acarida syndrome
Track 9: Rare Diseases in Cancer and Treatment
A rare cancer might be a subtype of a most common cancer. Rare cancers include thyroid cancer, acute lymphoblastic leukemia (ALL) and mouth cancer. There are also many rare cancers most people will not be familiar with. Although the incidence of individual rare cancers is low, as a collective they represent nearly a quarter of all cancers, more than any single common cancer. Rare cancers have been divided into two groups: cancers defined by their unusual histogenesis (cell of origin or differentiation state) and histologically defined subtypes of common cancers. The presence of specific genetic variants provides rationale for the testing of targeted drugs in rare cancers and they represent roughly 20% of all human cancers and are associated with worse survival. Diagnosis of rare cancers may consume a lot of time and about one in five rare cancers affect the blood and lymphatic system. Rare cancers caused by simple genetic mutations and common cancers tend to be caused by a complex set of genetic and epigenetic aberrations that continually grow in number as the tumor develops. A cancer is considered to be rare if-
The major types of cancer are
-
Carcinoma
-
sarcoma
-
melanoma
-
lymphoma
Track 10: Viral Infectious Diseases
Viral Infectious Disease occurs when an organism's body is invaded by pathogenic viruses, and infectious virus particles attach to and enter susceptible cells. There are plenty of viruses that cause a wide range of viral diseases. Viruses are very tiny germs. They are made of genetic material inside by a protein coating. Viral diseases are contagious and can spread from person to person when a virus enters the body and begins to multiply. Viral diseases result in a wide variety of symptoms that vary in character and severity depending on the type of viral infection and other factors, including the person’s age and overall health.
-
Bowenoidpapulosis
-
California encephalitis
-
Human T-cell leukemia virus
-
Congenital rubella
-
Kaposi sarcoma
Track 11: Sexually Transmitted Diseases
Sexually transmitted infections (STIs), also referred as sexually transmitted diseases (STD), are infections that are commonly spread by sexual activity, especially vaginal coition, anal sex or oral sex. Most STIs initially do not cause symptoms. This result causes a great risk of passing the disease on to others. STIs attained before or during birth may result in poor out comes for the baby. Some STIs may cause problems with the ability to get conceive. More than 30 different microbial pathogen can cause STIs. STIs usually spread by sex, some of them by non-sexual contact like infected blood tissues, breastfeeding, or during childbirth. STI diagnostic tests are easily available. Depending on the infection, untreated STIs can lead to infertility, chronic pain or even death. Viral STIs include genital herpes, HIV/AIDS, and genital warts among others
-
Parasitic STIs comprises trichomoniasis among others
-
Of the most common infections, syphilis, gonorrhea, chlamydia, are curable, while herpes, hepatitis B, HIV/AIDS, and HPV are treatable but not curable.
Track 12: Rare Mental and Behavioral Disorders
Abnormality is also called as dysfunctional behavior which is assigned to the behavioral characteristic to those conditions regarded as rare or dysfunctional. A Behavior is considered to be abnormal when it is atypical or unusual behavior; 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 conditions 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.
-
Apotemnophilia
-
Alien Hand Syndrome
-
Stendhal Syndrome
-
CapgrasSyndrome
-
Alice in Wonderland Syndrome
Track 13: Rare Oral Diseases
The oral Oral-facial-advanced syndrome is a broad 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 presentation of signs and symptoms is extremely varied, making the diagnosis difficult. OFDS type I (Papillon-Leage-Psaume syndrome) is the most common of all of these disorders, and it is quite rare. OFDS type I is associated with a mutation on the X chromosome of a gene designated CXORF5 and in most cases, it is believed, the mutation occurs randomly (sporadic). Other rare oral diseases include Odontoma dysphagia syndrome, Tricodento-osseous syndrome, Oral sub mucous fibrosis, Bam forth syndrome, Dentin dysplasia sclerotic bones. Undiagnosed diseases also are of prior importance. The symptoms of these diseases can be useful for the current researchers for their diagnosis.
-
Odontoma dysphagia syndrome.
-
Trichodento-osseous syndrome.
-
Oral sub mucous fibrosis.
-
Bam forth syndrome.
-
Dentin dysplasia sclerotic bones.
Track 14: Rare Skin Diseases
Advancements in research for the treatment of rare skin conditions with genetically modified stem cells have recently garnered widespread attention amongst the scientific community. Nonetheless, complications can arise with gene therapy, such as the overexpression of genes leading to tumor development. Despite the shortage of information relating to the adverse effects of the therapy on the patient, if deemed safe, this therapy could revolutionize the treatment of rare skin disorders. The rare skin diseases are most commonly seen in American countries and millions of people are affected by one or the other type of skin disorders. These skin diseases affect the protective barrier, that is, skin from performing its normal functions.
-
Argyria
-
Morgellons disease.
-
Vitiligo
-
Erythropoietin protoporphyria
-
Harlequin ichthyoids
-
Blau syndrome
-
Epidermolytic ichthyosis
Track 15: Rare Immunological Diseases
Immune system is a body defense system and works to defend the body against harmful foreign substances, or pathogens, either by preventing them from entering the body in the first place or by attacking them once they get inside. The immune system acts as a layer of defense and engulfs or swallows the foreign substances entering the body. The changes in the activities of the immune system may lead to reduced immunity. In a rare condition, like in SCID, Severe Combined Immunodeficiency, the infant is born without a developed adaptive immune system. This disease is expected to occur in 1 of 100,000 births. The child is extremely responsive to infection and various other diseases. Some of the other diseases that affect the immune system: Chediak-Higashi Disease, Mosaic variegated aneuploidy (MVA) syndrome, hereditary angioedema with C1-inhibitor deficiency, Warts Hypo-gamma-globulinemia Immunodeficiency Myelokathexis (WHIM) syndrome, Bradykinin-mediated angioedema (Bk-AE) and many more.
-
RPI deficiency
-
Fields Condition
-
Kuru
-
Methemoglobinemia
-
Hutchinson-Gilford Progeria
Track 16: Allergic and Immunologic Disorders
Allergy is one of the adverse immune reaction which is next accompanied by inflammation. The set of reactions 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 immune system is delegated to defend the body from attacks outside or inside. Many of the diseases that target immune system and reducing its ability to defend self or the fighting ability against foreign cells or inducing an abnormal response against external or internal antigens. Many diseases can affect immune system reducing its ability to defend self or inducing an abnormal response against external or internal antigens.
-
Allergic Rhinitis (Allergies) Allergic rhinitis is an allergic reaction to inhaled substances.
-
Angioedema. Angioedema is swelling under the skin, rather than on the skin as is the case with hives
-
Asthma
-
Atopic Dermatitis (Eczema)
-
Autoimmune Disorders
-
Bronchitis
-
Celiac Disease
Track 17: Rare Ophthalmology Diseases
Ophthalmological Diseases in which rare, hereditary vision disorder within which an individual has frequent or no ability to visualize color. Since ophthalmologists perform an operation to eyes, and also involving with both surgical and medical specialists to treat the rare conditions in eyes, some of them are receiving treatments. A large number of diseases and conditions may be diagnosed with rare eye problems affecting everyone. Individuals with achromatopsia conjointly usually experience some vision loss, especially within the brightest light, to which they extremely cause vision impairment. The severity of the illness achromatopsia varies differently. Although there'll be no cure or treatment for this disorder type, individuals with rare Ophthalmological conditions will manage their symptoms. The main factor they get involved as they will wear spectacles or tinted contact lenses to address bright light. They'll use magnifiers and different devices for low power vision to assist them read, and telescopes to assist them see distant objects
-
Behçet's disease of the eye.
-
Bietti's crystalline dystrophy.
-
Coloboma.
-
Idiopathic intracranial hypertension.
-
Microphthalmia (missing or small eyes)
-
Retinitis pigmentosa.
-
Retinoblastoma
Track 18: Genitourinary Rare Diseases
A number of rare diseases affect the kidneys and the urinary system. Some of these diseases are confined only to the urinary system and some are them also show symptoms in the different organ systems. The severity of the diseases varies affecting different parts of the urinary system like urinary tracts, tubules, glomerulus, Bowman’s capsule of the nephron, etc. Some diseases also may cause due to inflammation and autoimmune disease. The vasculature of the kidneys also gets affected, causing improper filtration of the urine from the blood vessels. The blood cells get excreted out in the form of urine. The most widely seen rare conditions in the genitourinary system are Emphysematous cystitis, urofacial syndrome, Fowler's syndrome, and glomerulonephritis, etc.
-
Amyopathic dermatomyositis.
-
Congenital erythropoietin porphyria.
-
Cranioectodermal dysplasia.
-
Erytoietic protoporphyriahrop.
-
Multicentric osteolysis nephropathy.
Track 19: Rare Hematological Diseases
When the normal process of blood cell growth and development breaks down, certain cell types can be produced in abnormally large quantities or in abnormal form which gives rise to blood cancers or other blood disorders. There are two types of rare blood diseases, they include: Myeloproliferative disorders also referred as myeloproliferative neoplasms (MPNs) and Lymph proliferative disorders also referred as lymph proliferative neoplasms (LPNs). Myeloproliferative disorders originate when the bone marrow starts making too many red blood cells, platelets, or certain white blood cells whereas, lymph proliferative disorders.MPNs include: Polycythemia Vera, Myelofibrosis, Essential thrombocythemia, Eosinophilia, Mastocytosis, Histiocytosis and Paroxysmal nocturnal hemoglobinuria. LPNs include: Hairy cell leukemia (HCL) and Large Granular Leukemia (LGL).
-
Myelofibrosis
-
Essential thrombocythemia
-
Eosinophilia
-
Mastocytosis
-
Histiocytosis
-
Paroxysmal nocturnal hemoglobinuria (PNH)
Track 20: Rare Pulmonary Diseases
Respiration involves gas exchange in higher organisms. Respiratory disorders occur in the respiratory tract, which includes the alveoli, bronchi, bronchioles, pleura, pleural cavity, trachea and the nerves and muscles of breathing. Some of the rare pulmonary disorders include: lymphangioleiomyomatosis, Pulmonary Alveolar Proteinases, Pulmonary Langerhans Cell Histiocytosis, Pulmonary Alveolar Microlithiasis, Pulmonary vacuities, Idiopathic eosinophilia pneumonias and many more. The common symptoms of the above-mentioned disorders are: breathlessness, swollen lymph nodes, chest pain, fatigue etc. The cost of treatment for some rare diseases is very expensive.
-
Idiopathic interstitial pneumonias.
-
Hypereosinophilic lung diseases.
-
Allergic bronchopulmonary aspergillosis (ABPA)
-
Pulmonary vasculitis.
-
Alveolar hemorrhage syndromes.
-
Pulmonary lymphangioleiomyomatosis (LAM)
-
Alveolar proteinosis.
Track 21: Clinical Case Report on Rare diseases
Clinical Case Report is a detailed report of various symptoms, signs, diagnosis and treatment, and also a follow-up of an individual patient. Major scientific breakthroughs ensuing from investigation of rare diseases have usually provided insight into a lot of common disorders. The satisfaction of diagnosis a patient with a rare disorder with success is commonly chop-chop countered by the conclusion that the flexibility to know and treat the patient’s condition is restricted by mental object and also the difficulties of finding out the malady. Maybe the foremost frequent downside is that the enlisting of a requisite variety of study subjects for Associate in Nursing data-based cohort or a run. This would like needs the creation of multi-institutional and international collaborations to conduct clinical investigation in rare diseases
Track 22: Clinical Research on Orphan Drugs
A rare disease is any disease that affects a small percentage of the population. This research provides opportunities to study on rare diseases. Major scientific breakthroughs resulting from investigation of rare diseases have regularly given knowledge into more common disorders. The satisfaction of diagnosing a patient with a uncommon disorder successfully is often rapidly countered by the realization that the capacity to understand and treat the patient’s condition is limited by ignorance and the difficulties of studying the disease. Additionally, for the “interesting” patient with an uncommon disease, being a carcinoma to physicians may intensify suffering.
Track 23: Orphan Medicinal Products
Orphan drugs are medicinal products that are used to treat rare diseases. They are called “orphan drugs” because due to their limited in market, only few pharmaceutical companies pursue research into such products. These drugs would not be produce without government assistance.
List of orphan medicinal products
-
Adcetris
-
Adempas
-
Hetlioz
-
ChenodeoxycHolicAcidLeadiant
-
Besponsa
-
Iclusig
-
Ketoconazlehra
-
Namuscla
-
Roxon
Market Research Growth on Rare Diseases and Orphan Drugs
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 by the FDA and brought in to the market. Rare diseases are rare and often debilitating or even life-threatening diseases or conditions with a prevalence of 0.65%-1%, the cause of rare diseases is mostly genetic in nature and sometimes it may be as a result of infections or degenerative causes.
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 the therapeutic area, which is 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 2022.
Market Report of Orphan Drugs in European region
Between 5,000 and 8,000 distinct rare diseases exist, affecting between 6% and 8% of the population in total – in other words, between around 27 million and 36 million people in the EU. Most people suffer from diseases affecting fewer than 1 in 100,000 people.80% of rare diseases have identified genetic origins, and affect between 3% and 4% of births. Other rare diseases are due to degenerative and proliferative causes. Symptoms of some rare diseases may appear at birth or in childhood, including spinal muscular atrophy, lysosomes storage disorders, patent duct us arteriosus (PDA), familial adenomatous polyposis (FAP) and cystic fibrosis. More than half of rare diseases appear during adulthood, such as renal-cell carcinoma and acute myeloid leukemia.
Target Audience
-
Professors
-
Specialists
-
Researchers
-
Students
-
Industrial delegates
-
Academia and Research
-
Scientists
-
Biomedical companies
-
Healthcare Professionals
-
Infectious Diseases Specialist
-
Directors, Board Members, Presidents, Vice Presidents, Deans and Head of the Departments Scientists
-
Rare Diseases Associations and Societies
-
Patient Group
-
Business Entrepreneurs and Industrialists
Why to attend?
Join the Rare Diseases and Orphan Drugs Congress to keep up to date with the industry and to learn from our speaker panel, bringing you important new case studies, people sharing their research views and reports on these relevant topics.
-
Gain knowledge of different rare diseases and their unique challenges, as well as how treatment methods can be transferred 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
-
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
-
These diseases are responsible for rising healthcare costs, lost productivity, and long-term disability.
-
It is a chance to communicate with individuals over the globe concentrated on finding out about Rare diseases and orphan drugs.
Scope and Importance of Rare Diseases and Orphan Drugs
Rare diseases
A rare disease is any disease that affects a small percentage of the population. In some parts of the world, an orphan disease is a rare disease whose rarity means there is a lack of a market large enough to gain support and resources for discovering treatments for it, except by the government granting economically advantageous conditions to creating and selling such treatments.
Most rare diseases are genetic and thus are present throughout the person's entire life, even if symptoms do not immediately appear. Many rare diseases appear early in life, and about 30% of children with rare diseases will die before reaching their fifth birthday
Orphan drugs
An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases.“Orphan drugs" are medicinal products intended for diagnosis, prevention or treatment of life-threatening or very serious diseases or disorders that are rare.
Major Associations Worldwide
· National Organization for Rare Diseases
· Canadian Organization for Rare Diseases
· Organization for Rare Diseases India
· The Boler-Parseghian Center for Rare & Neglected Diseases
· Cystic Fibrosis Foundation
·European Union Committee of Experts on Rare Diseases
· Multiple Myeloma Research Foundation
· U.S. Food and Drug Administration
·Birmingham children’s Hospital
·EURORDIS
·Every Life Foundation for Rare Diseases
·Global Genes
·Rare Diseases South Africa
. Short Bowel Syndrome Foundation
. Rare Disorders Society Singapore
· Genetic and Rare Diseases Information Center (GARD)
· Students4RareDiseases
· Malaysian Rare Disorders Society
· New Zealand Organization for Rare Disorders (NZORD)
·Rare Disease United Foundation
·Rare Diseases Denmark
· Genetic Alliance UK
·Rare Voices Australia
·BioPontis Alliance for Rare Diseases
. Rare Diseases Malta
· The International Rare Diseases Research Consortium (IRDIRC)
· Genetic Diseases Association - UAE
· National Tay-Sachs and Allied Diseases (NTSAD)
Top Universities Worldwide
· Harvard University
· University of Oxford
· University of Cambridge
· Rare Genomics Institute
· GMEC, The Global Medical Excellence Cluster
· University of Zurich
· Stanford University
· Cambridge University
· Yale University
· Emory University
· Karolinska University
· John Hopkins University
· Newcastle University
· University of Valencia
· Osaka University
·McMaster University
Hospitals Associated with Rare Diseases Research Worldwide
·The Manton Center for Orphan Disease Research (Boston, USA)
·US hospital for rare disease research (USA)
·NORD- National Organization for Rare Disorders (USA)
. Chicago Rare Disease Foundation (Chicago, USA)
· National Institute of Health (NIH) funds research consortia to study more than 200 rare diseases (USA)
. Children’s Hospital of Pittsburgh (Center for Rare Disease Therapy) (USA)
·Birmingham children’s Hospital (NHS Foundation Trust) (UK)
·The Children’s Hospital of Philadelphia (USA)
Industries Associated with Rare Diseases Research Worldwide
. Abbvie
· Actelion
· AFM-Telethon
· Alexon
· Amgen
· Amicus Therapeutics
. AstraZeneca
· Baxter
· Bayer
· Biogen
· Bio Marin
· Bioxcel
· Bristol-Myers Squibb
· Celgene
· Cyan
· Eli Lilly
·Genentech
· Gilead Sciences
·GlaxoSmithKline
·Johnson & Johnson
· Leadiant Biosciences
· Lumos Pharm
· Medivir
· Merck
· Novartis
· Novo Nordisk
. Orphan Swiss
·Orphan Europe
·orphan reach
·Pfizer
· Pharmacyclics
· Pharm Amar
· Pharm
·Prom etic Life Sciences
· Recordation Spa.
· Roche
· Sanofi Genzyme
. Sarepta Therapeutics
· Shire
· Spectrum Pharmaceuticals
· Swedish Orphan Biovitrum
· Takeda
· Teva
· Ultragenyx
· Vertex Pharmaceuticals
· Quintile Sims
