Hutchinson-Gilford progeria syndrome is a genetic condition characterized by the rapid, dramatic appearance of ageing in children. This condition is caused by a mutation in the lamin A (LMNA) gene. The affected children develop a characteristic facial appearance, including prominent eyes, small chin, protruding ears, thin lips and a thin nose with a beaked tip. Moreover, this syndrome leads to hair loss (alopecia), joint abnormalities, aged-looking skin, and a loss of fat under the skin (subcutaneous fat). Individuals suffering from Hutchinson-Gilford progeria syndrome are known to experience severe hardening of the arteries (arteriosclerosis) since early childhood. The condition worsens with growing age and thus, escalates the risk of heart attack or stroke at a young age.
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Hutchinson-Gilford progeria is a rare condition known to affect at least one in around four million newborns across the world, as surveyed by the National Institutes of Health (NIH). Until now, around 130 cases have been reported as per the NIH statistics. The affected patients live up to 30 years maximum, with an average life span of 13 years. Nearly 90% of the patients die from complications related to atherosclerosis. Until 2012, there were no effective treatment options discovered for this syndrome. However, the available treatment options mostly focused on reducing cardiovascular symptoms and growth abnormalities.
Farnesyltransferase Inhibitor (FTI)
In 2012, findings of the first clinical trial of the drug Lonafarnib, a farnesyltransferase inhibitor (FTI), offered a new hope for the treatment of children ailing from Hutchinson-Gilford progeria syndrome. The clinical trial results demonstrated massive improvement in weight gain, increase in bone mineral density, reduced vascular stiffness, and improved sensorineural hearing in patients with progeria. Previous treatments with growth hormone, and Sulforaphane helped in reducing the symptoms, and prolong a child’s life. However, it is essential that the patient regularly visits the cardiologist. Rapamycin is one other drug used before, that demonstrated to reverse nuclear blebbing, retard cellular senescence, and facilitate degradation of progerin.
In 2015, a team of scientists at the Agency for Science, Technology & Research (A*STAR) successfully established a model of Hutchinson-Gilford progeria syndrome market. The study conducted by this organization proposed a model implying that progerin is linked to telomeres. Progerin induces a reduction in heterochromatin, a tightly packed form of DNA, making telomeres in the cell more fragile and susceptible to damage. The damaged telomeres in turn trigger premature cellular aging. This model was radically different from the previous one. The gene progerin caused the nucleus to be deformed, thereby weakening the ability of the cells to divide and proliferate. The altered progerin protein makes the nuclear envelope unstable and progressively damages the nucleus, making cells more likely to die prematurely.
Researchers are currently working on determining how genetic changes further lead to the significant characteristic features of Hutchinson-Gilford progeria syndrome. Continuous advancement in research, along with augmented understanding of the human aging, is expected to provide valuable insights pertaining to the condition, and therefore majorly aid in its treatment.
Research & development activities carried out by major institutes, in order to find novel therapies for age-related conditions is expected to offer lucrative opportunities for the global market players. For instance, in August 2019, researchers from the Houston Methodist Research Institute at the Texas Medical Center are focused on using RNA therapeutics—treatment that is focused on ribonucleic acids, a substance found in all living cells—to slow, and possibly reverse Hutchinson-Gilford Progeria. Similarly, in July 2019, a group of researchers from the University of Oviedo, Spain, established that fecal microbiota transplants can help prematurely old mice live longer. The research may help in designing the targeted probiotic treatments for age-related conditions, such as Hutchinson-Gilford Progeria in humans. Furthermore, in March 2019, researchers at the Centro Nacional de Investigaciones Cardiovasculares (CNIC), together with Universidad de Oviedo researchers identified a new molecular mechanism involved in the premature development of atherosclerosis in mice with Hutchinson-Gilford progeria syndrome. The newly identified therapeutic target could be used for blocking early atherosclerosis in progeria.
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Table of Content
Global Hutchinson-Gilford Progeria Treatment Market Research Report
Section 1: Global Hutchinson-Gilford Progeria Treatment Industry Overview
Section 2: Global Economic Impact on Hutchinson-Gilford Progeria Treatment Industry
Section 3: Global Market Competition by Industry Producers
Section 4: Global Productions, Revenue (Value), according to Regions
Section 5: Global Supplies (Production), Consumption, Export, Import, geographically
Section 6: Global Productions, Revenue (Value), Price Trend, Product Type
Section 7: Global Market Analysis, on the basis of Application
Section 8: Hutchinson-Gilford Progeria Treatment Market Pricing Analysis
Section 9: Market Chain, Sourcing Strategy and Downstream Buyers
Section 10: Strategies and key policies by Distributors/Suppliers/Traders
Section 11: Key Marketing Strategy Analysis, by Market Vendors
Section 12: Market Effect Factors Analysis
Section 13: Global Hutchinson-Gilford Progeria Treatment Market Forecast
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