Hutchinson-Gilford Progeria Syndrome (HGPS) Treatment: Introduction
Hutchinson-Gilford progeria syndrome (HGPS), is a rare, fatal, genetic condition in children with striking features resembling premature aging. Children with progeria usually have a normal appearance in early infancy. At approximately nine to 24 months of age, affected children begin to experience profound growth delays, resulting in short stature and low weight.
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They also develop a distinct facial appearance, characterized by a disproportionately small face in comparison to the head, an underdeveloped jaw, malformation and crowding of the teeth, abnormally prominent eyes, a small nose, and a subtle blueness around the mouth. Furthermore, by the second year of life, the scalp hair, eyebrows, and eyelashes are lost (alopecia), and the scalp hair may be replaced by small, downy, white or blond hair
Key Drivers of Global Hutchinson-Gilford Progeria Syndrome (HGPS) Treatment Market
The global HGPS treatment market is anticipated to expand rapidly in the near few years. Introduction of novel therapies and increase in cases of HGPS are likely to be major drivers of the Hutchinson-Gilford Progeria Syndrome (HGPS) treatment market.
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According to the National Organization for Rare Disorders (NORD), the prevalence of HGPS is approximately 1 in 20 million; therefore, at any given time, approximately 400 children are living with progeria worldwide. Use of genetic testing has been increasing substantially in the last few years, thereby positively influencing the genetic disease diagnosis market In November, 2020 the U.S. Food and Drug Administration approved Zokinvy (lonafarnib) capsules to reduce the risk of death due to Hutchinson-Gilford progeria syndrome and for the treatment of certain processing-deficient progeroid laminopathies in patients one year of age and older; the first-ever treatment for Progeria. Zokinvy, a farnesyltransferase inhibitor, is an oral medication that helps prevent the buildup of defective progerin or progerin-like protein.
Hospital Pharmacies to be Highly Lucrative Segment
Based on distribution channel, the global Hutchinson-Gilford Progeria Syndrome (HGPS) Treatment market can be categorized into retail pharmacies, hospital pharmacies, online pharmacies, and others The hospitals segment is anticipated to account for a major share of the market in the next few years due to a rise in the number of treatments in hospitals
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