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A rare disease is a disease that affects a small percentage of the population. In some parts of the world, the term orphan disease describes a rare disease whose rarity results in little or no funding or research for treatments, without financial incentives from governments or other agencies. Orphan drugs are medications targeting orphan diseases.
(Reuters) -The U.S. Food and Drug Administration has approved Zevra Therapeutics' drug for a rare and fatal genetic disorder, making it the first treatment to get a nod for the condition, the ...
Dr. Aaron Glatt, chief of infectious diseases at Mount Sinai South Nassau in Oceanside, New York, described AFM as "a rare but potentially very serious illness that can mimic polio and can cripple ...
Auto-brewery syndrome (ABS) (also known as gut fermentation syndrome, endogenous ethanol fermentation or drunkenness disease) is a condition characterized by the fermentation of ingested carbohydrates in the gastrointestinal tract of the body caused by bacteria or fungi. [1] ABS is a rare medical condition in which intoxicating quantities of ...
Fibrodysplasia ossificans progressiva (/ ˌ f aɪ b r oʊ d ɪ ˈ s p l eɪ ʒ (i) ə ɒ ˈ s ɪ f ɪ k æ n z p r ə ˈ ɡ r ɛ s ɪ v ə /; [1] abbr. FOP), also called Münchmeyer disease or formerly myositis ossificans progressiva, is an extremely rare connective tissue disease in which fibrous connective tissue such as muscle, tendons, and ligaments turn into bone tissue (ossification).
Frequency. around 1 in 500,000 [5] Epidermolysis bullosa (EB) is a group of rare medical conditions that result in easy blistering of the skin and mucous membranes. Blisters occur with minor trauma or friction and are painful. Its severity can range from mild to fatal. [7] Inherited EB is a rare disease with a prevalence in the United States of ...
This condition is inherited in an autosomal dominant manner. Blepharophimosis, ptosis, epicanthus inversus syndrome (BPES) is a rare medical anomaly characterized by the conditions it is named after: blepharophimosis, ptosis and epicanthus inversus. There are two types; type 1 is distinguished from type 2 by including the symptom of premature ...
Foreign accent syndrome is a rare medical condition in which patients develop speech patterns that are perceived as a foreign accent [ 1 ] that is different from their native accent, without having acquired it in the perceived accent's place of origin. Foreign accent syndrome usually results from a stroke, [ 1 ] but can also develop from head ...