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Showing posts with the label Enzyme Replacement Therapy

Lysosomal Storage Diseases Therapeutics are getting better with continuous R&D activities among key pharmaceutical companies

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  Lysosomal Storage Disease is a medical condition where proteins, usually DNA, become deformed or lose their catalytic ability. The human genetic material, which is called DNA, is a highly dynamic and sophisticated part of our system. Due to a process called mutations, DNA becomes altered and either changes its directions or becomes a different size. In either case, the genetic information is then unreadable by cells known as enzymes. There are various types of lysosomal storage diseases including Gaucher disease, Fabry disease, Pompe disease, Niemann-Pick disease, Tay-Sachs disease, etc. Lysosomal storage diseases therapeutics aim at correcting these mutations so that the DNA can be read again by cells. Since the function of the human body is to produce new cells, any change in DNA is extremely dangerous. People with lysts have had to live with the lifelong threat of illness because even minor changes in their DNA structure could lead to serious illness. The human population a...

Hunter Syndrome is a condition in which a person The patient's age and the seriousness of the disease determine the course of treatment

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  Hunter syndrome treatment incorporates directing indications and intricacies as there is no solution for the ailment. Hunter syndrome, in any case called mucopolysaccharidosis type II, is an inherited issue wherein colossal sugar particles called glycosaminoglycans create in body tissues. It is an extraordinary innate issue achieved by a missing or bombing compound that impacts various bits of the body. This prompts inadequacy of a protein called iduronate-2-sulfatase, similarly as to issues during the bones, respiratory structure, heart, and various organs. Hunter syndrome is of two sort, as delicate and genuine. Impacted individuals by and large fail horrendously during their young adult years. Hunter syndrome is an auto-safe issue known to impact the two children and adults. All around, it begins in the second year of life. In infants, it shows as help cap; while in adults, it shows as sensitive palmar hyperhidrosis, which makes the patient sweat in the armpits. In specific ...

Hunter Syndrome Treatment is based on the patient's age and severity of the disease

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  Hunter syndrome treatment includes overseeing manifestations and complexities as there is no remedy for the sickness. Hunter syndrome, otherwise called mucopolysaccharidosis type II, is a hereditary issue wherein enormous sugar particles called glycosaminoglycans develop in body tissues. It is an uncommon hereditary problem brought about by an absent or failing chemical that influences numerous pieces of the body. This prompts insufficiency of a protein called iduronate-2-sulfatase, just as to issues during the bones, respiratory framework, heart, and different organs. Hunter syndrome is of two sort, like gentle and serious. Influenced people generally bite the dust during their adolescent years. Hunter syndrome is an auto-safe problem known to influence the two kids and grown-ups. By and large, it starts in the second year of life. In babies, it shows as support cap; while in grown-ups, it shows as delicate palmar hyperhidrosis, which makes the patient perspiration in the armp...