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Migalastat, sold under the brand name Galafold, is a medication for the treatment of Fabry disease, a rare genetic disorder. It was developed by Amicus Therapeutics . The US Food and Drug Administration (FDA) granted it orphan drug status in 2004, [ 5 ] and the European Commission followed in 2006. [ 6 ]
Fabry disease, also known as Anderson–Fabry disease, is a rare genetic disease that can affect many parts of the body, including the kidneys, heart, brain, and skin. [1] Fabry disease is one of a group of conditions known as lysosomal storage diseases .
Pegunigalsidase alfa, sold under the brand name Elfabrio, is an enzyme replacement therapy for the treatment of Fabry disease. [2] [4] It is a recombinant human α-galactosidase-A. [4] It is a hydrolytic lysosomal neutral glycosphingolipid-specific enzyme. [2] The most common side effects are infusion-related reactions, hypersensitivity and ...
Enzyme replacement therapy is available to treat mainly Fabry disease and Gaucher disease, and people with these types of sphingolipidoses may live well into adulthood. The other types are generally fatal by age 1 to 5 years for infantile forms, but progression may be mild for juvenile- or adult-onset forms.
The bacterial Shiga toxin can be used for targeted therapy of certain gastrointestinal cancers that express the receptor of the Shiga toxin. [4] For this purpose a non-specific chemotherapeutic agent is conjugated to the B-subunit to make it specific. In this way only the tumor cells, but not healthy cells, should be destroyed during therapy. [5]
This list features both the added and removed subtypes. Also, 22 ICD-9-CM codes were updated. [2] The ICD codes stated in the first column are those from the DSM-IV-TR. The ones that were updated are marked yellow – the older ICD codes from the DSM-IV are stated in the third column.
The DSM-5 (2013), the current version, also features ICD-9-CM codes, listing them alongside the codes of Chapter V of the ICD-10-CM. On 1 October 2015, the United States health care system officially switched from the ICD-9-CM to the ICD-10-CM. [1] [2] The DSM is the authoritative reference work in diagnosing mental disorders in the world.
This is an accepted version of this page This is the latest accepted revision, reviewed on 21 January 2025. The following is a list of mental disorders as defined at any point by the Diagnostic and Statistical Manual of Mental Disorders (DSM) or the International Classification of Diseases (ICD). A mental disorder, also known as a mental illness, mental health condition, or psychiatric ...