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Rare diseases are the diseases or the condition of that particular member that affect fewer people. The treatment of these conditions has been orphaned or discontinued the reason of there is not financial support for the development of the drug. So, these are the drugs that are not developed by the pharma industry but respond to public health.
The orphan drugs are medical products that are developed for the treatment of rare diseases. A rare disease is a condition that affects a small percentage of the population.
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Orphan Drugs & Rare Disease Innovations: Explores the discovery, development, and clinical application of orphan drugs for rare diseases. It covers innovative therapeutic strategies, research methodologies, and the translation of scientific discoveries into effective treatments. Participants will gain insights into cutting-edge advancements that improve patient care and expand treatment options for rare conditions.
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Clinical case reports and case studies are important tools in medical research and education. They provide detailed descriptions of individual patients' medical histories, symptoms, diagnostic evaluations, treatments, and outcomes.
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Epidemiology and Disease Control: Epidemiology and disease control in rare conditions involve studying distribution, frequency, and underlying causes of uncommon illnesses. This helps identify risk patterns, support prevention strategies, and inform public health planning.
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They are different types of rare diseases that are based on the different body parts, different diseases, different gene, etc
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Public Health Perspectives in Rare Diseases: Public health perspectives in rare diseases emphasize policies, infrastructure, social support systems, and community strategies that ensure equitable access to diagnosis, treatment, and long-term care for individuals living with rare conditions.
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AI in Rare Diseases and Orphan Drugs: Artificial Intelligence (AI) in rare diseases and orphan drugs refers to the use of advanced computational methods to identify disease patterns, predict treatment response, accelerate drug discovery, and support clinical decision-making. AI helps overcome limited patient data, enabling faster diagnosis and more targeted therapeutic development.
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Clinical Trial Design for Rare Conditions: Clinical trial design for rare conditions focuses on creating study formats that work with small patient populations, diverse clinical presentations, and limited historical data. These designs aim to generate reliable evidence while ensuring patient safety and practical feasibility.
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Infectious diseases and immune deficiencies are closely related aspects of human health that involve complex interactions between pathogens and the immune system. Understanding the mechanisms underlying these conditions is essential for developing effective strategies for prevention, diagnosis, and treatment, as well as for improving overall public health and patient outcomes.
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Rare Diseases: Treatments & Therapies: Treatment of rare diseases involves a range of approaches, including conventional therapies, advanced medical interventions, and innovative treatments such as gene therapy or targeted drug therapy. Effective management requires personalized care plans, ongoing research, and collaboration among healthcare professionals to improve patient outcomes and offer hope for better quality of life
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Genetics and Genomics of Rare Diseases: Genetics and genomics of rare diseases focus on identifying DNA variants, gene mutations, and molecular abnormalities responsible for rare disorders. Genomic tools help clarify disease mechanisms, improve diagnosis, and guide precision treatments.
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Rare Diseases: Causes, Challenges & Diagnosis: Rare diseases are medical conditions that affect a small percentage of the population, often caused by genetic mutations, environmental factors, or unknown origins. These conditions present unique challenges for patients, including delayed diagnosis, limited treatment options, and physical, emotional, and social difficulties. Accurate and early diagnosis is crucial to managing symptoms, providing appropriate care, and improving quality of life.
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Rare Hematologic Disorders: Rare hematologic disorders are uncommon conditions affecting the blood, bone marrow, or clotting systems. They often require specialized diagnostic tools and tailored treatment due to their complex and variable clinical behavior.
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Rare neurological disorders encompass a diverse group of conditions that affect the nervous system and typically have a low prevalence in the population. These disorders may involve the brain, spinal cord, nerves, or muscles, leading to a wide range of neurological symptoms and impairments. While each rare neurological disorder is unique, many shares common features such as genetic predisposition, progressive degeneration, and significant impact on quality of life.
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Rare autoimmune disorders encompass a diverse group of conditions in which the immune system mistakenly attacks the body's own tissues, leading to inflammation, tissue damage, and dysfunction. While some autoimmune disorders are relatively common, there are also rare autoimmune diseases that affect a small percentage of the population.
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Rare nutritional and metabolic diseases encompass a diverse group of disorders that affect the body's ability to metabolize nutrients, leading to deficiencies, toxicities, or abnormalities in metabolic processes. These conditions often result from genetic mutations or defects in enzymes, receptors, or transport proteins involved in nutrient metabolism.
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Pediatric rare diseases are uncommon medical conditions that specifically affect children, often beginning early in life. These diseases are typically defined as "rare" because they affect a very small percentage of the population—generally fewer than 200,000 people in total across the U.S., though criteria can vary by country.
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Rare skin diseases are uncommon conditions that affect the skin, often with distinctive symptoms and complex treatment needs. These diseases may result from genetic mutations, autoimmune disorders, infections, or unknown causes. Many rare skin diseases can impact not only the skin but also other organs and systems in the body
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Rare Neuromuscular Disorders: Rare neuromuscular disorders are a group of uncommon diseases that affect the nerves and muscles responsible for movement, coordination, and body control. These disorders interfere with the communication between the nervous system (neurological component) and the muscles (muscular component), leading to weakness, fatigue, muscle wasting, and loss of motor function.
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The rare syndrome I Covid-19 is defined as the multisystem infection syndrome appears in the children in a rare condition that associated with COVID-19. It appears 2 to 6 weeks after the child develops it involves the inflammation of the several body parts including the heart. Multisystem infection syndrome in the children is the rare condition that associated with the COVID-19 they can cause different internal and the external causes of the body parts to inflamed that includes heart, kidneys, eyes, brain, skin, etc.
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Rare cancers are malignancies that affect a small percentage of the population and often have limited research, treatment options, and awareness compared to more common cancers. These cancers can arise in various organs and tissues throughout the body and may present unique challenges in diagnosis, treatment, and management
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X-linked disorders primarily affect males due to their XY chromosome makeup, mitochondrial disorders can affect both males and females, although the severity and presentation may vary. Both types of disorders can present significant challenges in diagnosis, management, and treatment due to their genetic complexity and the wide range of symptoms they can cause.
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