Farber's disease - Epidemiology Insight - 2030
発行: DelveInsight Business Research LLP
ページ情報: 英文 66 Pages
DelveInsight's 'Farber's disease- Epidemiology Forecast-2030' report delivers an in-depth understanding of the Farber's disease, historical and forecasted epidemiology in the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.
Farber's disease: Disease Understanding
Farber's disease Overview
Farber's disease is a rare inherited condition involving the breakdown and use of fats in the body. In affected individuals, lipids accumulate abnormally in cells and tissues throughout the body, particularly around the joints. The deficiency of an enzyme is called ceramidase resulting in the harmful accumulation of certain chemicals in the body, which causes damage and inflammation.
Three classic signs occur in Farber lipogranulomatosis: a hoarse voice or a weak cry, small lumps of fat under the skin and in other tissues (lipogranulomas), and swollen and painful joints. Affected individuals may also have difficulty breathing, an enlarged liver and spleen (hepatosplenomegaly), and developmental delay.
Researchers have described seven types of Farber lipogranulomatosis based on their characteristic features: Type 1 is the most common, or classical, form of this condition and is associated with the classic signs of voice, skin, and joint problems that begin a few months after birth. Developmental delay and lung disease also commonly occur. Infants born with type 1 Farber lipogranulomatosis usually survive only into early childhood.
Types 2 and 3 generally have less severe signs and symptoms than the other types. Affected individuals have three classic signs and usually do not have developmental delays. Children with these types of Farber lipogranulomatosis typically live into mid- to late childhood.
Types 4 and 5 are associated with severe neurological problems. Type 4 usually causes life-threatening health problems beginning in infancy due to massive lipid deposits in the liver, spleen, lungs, and immune system tissues. Children with this type typically do not survive past their first year of life. Type 5 is characterized by a progressive decline in the brain and spinal cord (central nervous system) function, which causes paralysis of the arms and legs (quadriplegia), seizures, loss of speech, involuntary muscle jerks (myoclonus), and developmental delay. Children with type 5 Farber lipogranulomatosis survive into early childhood. Due to the extremely progressive and severe nature of the disease, current treatments focus on symptom management and enhancing an individual's quality of life rather than completely eliminating symptoms. Corticosteroids are commonly used to relieve pain associated with inflammation of the joints. Some individuals require surgical procedures to restore proper breathing, which is impaired by abnormal tissue growth in the airway. Additionally, some individuals undergo cosmetic surgery to remove nodules and abnormal growths of the facial area.
The Farber's disease epidemiology division provides insights about the historical and current patient pool along with the forecasted trend for every seven major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the DelveInsight report also provides the diagnosed patient pool and their trends along with assumptions undertaken.
The disease epidemiology covered in the report provides historical as well as forecasted Farber's disease epidemiology segmented as the total diagnosed prevalent cases of Farber's disease, gender-specific cases of Farber's disease, and type-specific cases of Farber's disease. The report includes the Prevalent scenario of Farber's disease symptoms in 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom), and Japan from 2017 to 2030.
The epidemiology segment also provides the Farber's disease epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.
The total prevalent population of Farber's disease in the 7MM countries was estimated to be 86 cases in 2017.
As per the estimates, in 2017, the United States had the highest prevalent population of Farber's disease. Among the EU5 countries, Germany had the highest prevalent population of Farber's disease with 8 cases, followed by France in 2017. On the other hand, Spain had the lowest prevalent population in 2017.
DelveInsight interview KOLs and obtain SME's opinion through primary research to fill the data gaps and validate our secondary research. The opinion helps to understand the total patient population and current treatment pattern. This will support the clients in potential upcoming novel treatment by identifying the overall scenario of the indications.
Key Questions Answered
Farber's disease Epidemiology report will allow the user to:
Study Period: 2017-2030
The Farber's disease Research Consortium in the USA, the European Farber's disease Registry in Europe, and the Japanese Farber's disease Registry (JBAR) have been formed to help with data collection and provide a platform for large research studies.