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Substrate reduction therapy is FDA approved and there is at least one treatment available on the market. [10] Gene therapy aims to replace a missing protein in the body through the use of vectors, usually viral vectors. [11] In gene therapy, a gene encoding for a certain protein is inserted into a vector. [11]
Atidarsagene autotemcel, sold under the brand name Libmeldy among others, is a gene therapy treatment for metachromatic leukodystrophy developed by Orchard Therapeutics.It contains an autologous CD34⁺ cell enriched population that contains haematopoietic stem and progenitor cells transduced using a lentiviral vector encoding the human arylsulfatase A (ARSA) gene.
American Society of Gene & Cell Therapy (ASGCT) is a professional non-profit medical and scientific organization based in Milwaukee, dedicated to understanding, development and application of gene, related cell and nucleic acid therapies, as well as promotion of professional and public education on gene therapy. With more than 4,800 members in ...
Pancreatic enzymes, also known as pancreases or pancrelipase and pancreatin, are commercial mixtures of amylase, lipase, protease and lactase. [3] [4] They are used to treat malabsorption syndrome due to certain pancreatic problems. [3]
PTC Therapeutics’ therapy, known as PTC518, is an oral medication designed to reduce the production of the mutant protein linked to the genetic mutation responsible for the disease. [15] This mutant protein is believed to contribute to neuronal death, driving disease progression. [ 16 ]
Four different purified hyaluronidases have been approved for use in the United States, three of animal origin and one recombinant. They are indicated as adjuvants in subcutaneous fluid administration for achieving hydration, for increasing the dispersion and absorption of other injected drugs, or for improving resorption of radiopaque agents, in subcutaneous urography.