11^th International Conference on Rare Diseases & Orphan Drug November 05-05, 2024 Rome, Italy

Track 1 - Orphan diseases and public health

An orphan illness is one that affects fewer people than other diseases (such as cholera, typhoid, tuberculosis, and malaria) because it is significantly more widespread in underdeveloped countries than in the industrialised world. To identify the primary or secondary causes of health threats and then consistently prevent or reduce these causes in entire populations, public health practitioners use a combination of disciplines including basic science, epidemiology, behavioural research, clinical research, health care services, statistics, economics, and policy.

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

Track 2-Neglected Rare Tropical Diseases

Neglected tropical diseases (NTDs) include lymphatic  filariasis, dengue fever, leishmaniasis, and trachoma. They are named "neglected" because they primarily affect the poor and have historically received less attention than other diseases. They are a collection of tropical diseases that are especially common in low-income populations and developing countries such as Africa, Asia, and America. Some are relatively pricey, while others are incurable.  Tropical illness mortality have lately been studied, and they appear to be greater in low-income countries.

• Buruli Ulcer
• Chagas Disease
• Cysticercosis
•  Dengue Fever

Track 3-Orphan Drugs- Development Trends and Strategies

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

Track 4- Rare Obesity and Metabolic Disorders

Valinemia is an extremely rare metabolic disease. It is distinguished by high levels of the amino acid valine in the blood and urine caused by a valine transaminase enzyme deficiency. This enzyme is required for valine breakdown (metabolism). Obesity is similar to some major health hazards that can impact quality of life, such as type 2 diabetes, cardiovascular disease, and some types of cancer. Diabetes is one of the most frequent metabolic disorders that is caused by a hereditary component.

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

Track 5- Allergic and Immunologic Disorders

Allergies, often known as allergic illnesses, are a group of pathologies produced by the immune system's reactivity to common yon harmful materials in the environment. Food allergies, dermatitis, hay fever, atopic dermatitis, allergic asthma, and anaphylaxis are examples of these illnesses. Allergic disorders and its principal symptoms are caused by a developing immune system that reacts to normally harmless substances such as pollens and pet dander. They are easily diagnosed if we have good control over them.

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

Track 6- Paediatric rare diseases

The biopharmaceutical industry has made enormous progress in moving along with innovation for rare diseases; yet, some of the rare diseases do not have certain therapy alternatives, resulting in a significant unmet demand for patients. We've witnessed incredible progress in the development of medicines to treat patients with uncommon diseases as researchers uncover the molecular and genetic causes of many diseases. The deadly and hereditary disorders in children had striking similarities to accelerated ageing.

• Water allergy
• Foreign accent syndrome
• Laughing Death

Track 7- Future Hereditary of Orphan Drugs

A medicinal product delegated as an orphan medication is one that has been developed specifically to treat a rare medical problem, the condition described as "orphan disease" and drugs that are not manufactured of implementing by the pharmaceutical industry for economic reasons. Which acts as a public offer cause for families and individuals to assume the burden of uncommon diseases such as Batten Disease, Spinal Muscular Atrophy, and Duchenne.  The key reason for developing orphan drugs is to cure any future orphan rare diseases as well as any source of deficiency.

• RPI Deficiency
• Hutchinson-Gilford Progeria

Track 8- Mystery Diagnosis of Rare Diseases

The productive conversation about rare illness medicines has fallen short of offering vitally needed solutions for the vast majority of rare disease patients. Patients and clinicians frequently struggle to make a diagnosis due to neglectful symptoms, masquerade syndromes, scarcity of the disease or condition, or the patient's situation being an atypical presentation of condition or disease. As the programme partner in organising rare diseases has dubbed it.

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

Track 9- Rare Genetic Diseases

Due to a lack of connected clinical and diagnostic resources in the region, many suspected cases of genetic anomalies go untreated or misdiagnosed, leaving patients to face a significant psycho-socio-economic emergency and, in many cases, suffer long-term with their condition. The disorganisation that affects one child may differ significantly from the disorganisation that affects another. The most common cause of rare diseases is genetic growth, which is generated directly by chromosomes. It may also cause from one generation to the next in some cases.

• Polymelia
• Neurofibromatosis
• Diprosopus

Track 10- Rare Diseases of Genitourinary System

Many urinary system problems in children include acute and chronic kidney failure, urinary tract infections, urinary tract blockage, and birth defects. Kidney diseases can cause temporary or permanent alterations to the tiny functional structure and veins within the kidney. Intravenous Urography This test involves an X-ray of the urinary system. Before the X-rays are removed, a pigment is injected to make the urine visible on the X-ray, which can reveal any obstructions in the urinary tract or kidney problems.

• Acute Kidney Injury
• Alport Syndrome
• Nephropathic Cystinosis

Track 11-Viral Infectious Diseases and Sexually transmitted diseases

Sexually transmitted diseases (STDs) or sexually transmitted infections (STIs) are infections that spread from person to person through sexual contact. Contact is frequently made through sex. However, they can sometimes spread through additional intimate physical touch. This is because some STDs, such as herpes and HPV, are spread through skin-to-skin contact, which occurs naturally in the body. Human viruses are easily transmitted through sexual intercourse.

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

Track 12- Rare Oral Diseases and Rare Aging Diseases

To investigate how uncommon genetic illnesses influence skeletal development, socio-demographic variables, and oral health-related habits in children and adolescents using validated clinical measures. Rare genetic illnesses are frequently associated with additional co-morbid problems, which are frequently characterised by severe and chronically degenerative conditions associated with phenotypes2. Rare oral diseases can take many forms, including cancer, immune deficiencies, and metabolic disorders.

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

Track13- Orphan Drugs Treatment for Rare Diseases

medications developed expressly to treat rare diseases – known as "orphan drugs" – are frequently economically of Rare Disease On this day, it's worth revisiting a series of Healthcare Triage films we updated last year, which mostly focuses on orphan pharmaceuticals, what's wrong with the current system, and how we can get out of it. Can we, as a people, get out of this terrible system situation. An orphan drug is one that is used to treat orphan diseases, such as heam arginate and others. 

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

Track 14- Rare Bacterial, Viral and Fungal infections

Fungal illnesses are difficult to treat since the infection in the body spreads quickly. Mild fungal skin illnesses, which might resemble a rash, are extremely prevalent. Fungal diseases of the lungs are most similar to other conditions that can induce fever symptoms, such as bacterial or viral pneumonia. Some fungal diseases, such as fungal bloodstream infections and mucormycosis infections, can be fatal. Fingernails are the leading cause of fungal illnesses, and because we rarely wash our hands, the infection enters the mouth.

• Aspergillosis
• Blastomycosis
• Candidiasis

Track 15- Rare Endocrine Genetic Diseases

A rare disease is one that affects fewer people than is expected at any particular moment. According to the National Institutes of Health (NIH), among them are more unique uncommon disorders that typically impact the endocrine system. Their symptoms may appear rare in the body as ambiguous, making it difficult to pinpoint exactly what is wrong. Such "medical mysteries" frequently confound physicians, and patients visit doctor after doctor in search of answers with no definite diagnosis. As a result, people are dying at random.

• Ribose-5 phosphate isomerase
• Down Syndrome
• Thalassemia

Track16- Rare Gynaecological and Obstetrical Diseases

When children with a genetic syndrome reach adulthood, they may be referred to obstetrician-gynecologists for standard preventative or contraceptive services, screening or counselling for sexually transmitted infection, or menstruation management. Although some genetic diseases have no physical impairment, others do; thus, education and gynaecologic care should be dependent on a patient's psychological and physical skills. It is vital to note that teenagers, whether they have a genetic condition or not, are sexual creatures.

• Acute Abdomen and Pregnancy
• Adrenal Disease and Pregnancy

Track 17- Clinical Research and Public Awareness

Clinical research is critical to the NIH's aims of improving health, promoting health, and reducing the burden of sickness and disability. For example, clinical research can provide us with some insights and answers concerning the safety and efficacy of medications and other therapies, which we can then share with other researchers. To encourage patient patience and dedication, trial participants are encouraged to actively discuss their online experiences via social media, patient forums, or orally.

• Leveraging patient
• Volunteer advocacy

Treatment of Rare Diseases From 2019 to 2025, the market will increase significantly. The increasing prevalence of rare diseases around the world will drive growth in the rare disease treatment industry in the coming years. Rare diseases, often known as orphan diseases, afflict a small fraction of the population at any given moment. An orphan disease is one that affects fewer than 200,000 persons in the United States, according to the Food and Drug Administration (FDA). Under US legislation, the Orphan pharmaceuticals Act allows manufacturers to seek that the FDA confer orphan drug status to pharmaceuticals designed to treat a rare disease or condition.Market

Research Growth on Rare Diseases and Orphan Drugs:

There are about 7,000 different forms of Rare Diseases and Disorders, and it is believed that 30 million individuals in the United States, 30 million in Europe, and 350 million people worldwide suffer from them. Four-fifths of rare diseases are hereditary in nature, and half of those affected will be children. The Rare Diseases are dispersed in such a way that 350 Rare Diseases account for four fifths of all instances. Only around 5% of rare diseases have approved pharmacological treatment, with only 326 new treatments licenced by the FDA and brought to market.

Major Companies Associated with Rare Diseases and Orphan Drugs:

Orphan Drug sales were in the billions of dollars in 2018. Orphan drugs accounted for 35% of new drug approvals in the industry. The genetic illnesses are classified by therapeutic field, which led the worldwide market in the past and will continue to do so in the next eight years. By the end of 2025, this section is expected to be worth US$ 56,241. According to MRC statistics, the global orphan drug market was valued at $145.89 million in 2018 and is predicted to reach $265.63 million by 2022, rising at a CAGR of 10.5% between 2018 and 2025.

Related Associations and Societies

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

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

Thanks to all of our amazing keynote speakers, presenters, conference attendees, students, associations, media partners, and visitors Rare Diseases Meet 2023 was a huge success. The "10th International Conference on Rare Diseases & Orphan Drug" on December 23-24, 2023 at Dubai, UAE with the theme "Latest challenges for the eradication of rare diseases" was a big success thanks to Conference Series. The audience heard from renowned keynote speakers who represented a variety of prestigious organisations and groups. Renowned experts, members of the Editorial Board of journals in the Conference series, students, research scholars, scientists, young researchers, industrialists, Anaesthesiologists, doctors, Neurologists, and student researchers all generously and actively participated and helped make the event a success. A ceremony and speeches by the Keynote forum speakers marked the beginning of the conference. The experts who spread the message with their beautiful talk were. Let's reconnect @ Rare Diseases Meet 2024