Diseases

Autoimmune/Auto-inflammatory Disorders

Disease overviews, clinically relevant perspectives, and news about the most important research in rare autoimmune and auto-inflammatory disorders.
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Cancers

There are over 200 different types of rare cancers, but only a small number of people are diagnosed with each type. Many of those cancers are highlighted in this section.
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Congenital and Genetic Conditions

Congenital disorders can be inherited or caused by environmental factors and can vary from mild to severe. This section is focused on genetic and congenital disorders in children and adolescents.
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Endocrine Disorders

According to the NIH, there are more than 175 rare diseases that affect the endocrine system. In this section we highlight many of them and provide clinically relevant perspectives on these rare diseases.
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Gastrointestinal Diseases

Expert insight and general news into relevant research on diseases of the gastrointestinal tract.
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Heart Diseases

According to the CDC, as one in four deaths is a result of heart disease. Health care providers are trained to focus on the more prevalent heart diseases. This section is focused on rare and often neglected heart conditions.
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Hematologic Disorders

Many of the more “common” blood diseases are considered “rare,” affecting less than 200,000 U.S. citizens. These conditions primarily affect the blood & blood-forming organs. In this section we provide news, research summaries, and meeting reports relevant to the hematologic community.
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Kidney and Urinary Diseases

Diseases of the kidney and urinary affects over 30 million Americans and can lead to various related health problems, such as weak bones, nerve damage, and malnutrition. In this section we provide news and updates focused on rare kidney and urinary diseases.
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Lung Diseases

Learn about the symptoms, causes, management and available treatments of rare lung disorders. Perspectives on the latest clinically relevant research, guideline updates, and new drug approvals.
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Lysosomal Storage Disorders

Lysosomal storage diseases are a group of approximately 50 rare inherited metabolic diseases that are characterized by an abnormal build-up of various toxic materials in the body’s cells as a result of enzyme deficiencies. We provide interviews with leading experts, general news and clinical trial updates.
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Metabolic Disorders 

News, Interviews, and expert insight into the latest research relevant to rare metabolic diseases.
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Musculoskeletal Diseases

A wide range of rare conditions involve the musculoskeletal system. Some of these disorders arise from genetic problems in the muscles themselves, while others involve the nerves or bones.
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Neurology / Nervous System Diseases

Rare neurological disorders are diseases of the brain, spine, and autonomic nervous system. This section provides overviews of several of those conditions, as well news, clinical trial updates, and expert opinions form leading neurologists.
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Ophthalmology/Eye Diseases

Disease overviews and news and key expert videos focused on rare eye disorders.
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Skin Conditions

Disease overviews, clinically relevant perspectives, and news on the most important research in rare skin disorders.
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