Gitelman Syndrome

Sarepta Therapeutics reported updated results from a Phase II study, saying its experimental Duchenne muscular dystrophy (DMD) treatment SRP-5051 (vesleteplirsen) substantially increased dystrophin expression at the target dose. However, there were seven serious treatment-related adverse events involving cases of magnesium and potassium deficiency, although the company said these were well-managed.Billed as a next-generation version of Exondys 51 (eteplirsen), SRP-5051 is an investigational peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO) that binds exon 51 of dystrophin pre-mRNA. Monday's readout is derived from Part B of the MOMENTUM study, which enrolled 40 ambulant and non-ambulant DMD patients between the ages of 8 to 21 years old.

Bartter syndrome is a rare genetic disorder that causes specific defects in kidney function. Over the past few years, it is observed that different terminologies have been used to describe Bartter syndrome. Some classify Bartter syndrome on the basis of its clinical appearance, whereas others classify it on the basis of its underlying mutated gene. On the basis of the underlying gene mutation, Bartter syndrome can be classified as Bartter syndrome type 1, type 2, type 3, type 4a and 4b and Gitelman syndrome. Presently, according to the National Organization for Rare Disorders (NORD), Bartter syndrome is classified under a new classification system, which is based upon the specific part of the kidney affected. This includes loop disorders, DCT disorders and combined DCT and loop disorders. Loop disorders include Bartter syndrome type 1 and type 2 and DCT disorders include Gitelman syndrome. The diagnosis of Bartter syndrome includes laboratory tests such as blood tests for determining serum electrolyte levels, especially renin and magnesium and aldosterone levels, and urine tests for determining the presence of prostaglandin E2 and urine electrolytes. In addition, molecular genetic testing is also used for the diagnosis of Bartter syndrome. Molecular genetic testing detects the mutations in specific genes, which causes Bartter syndrome. However, molecular genetic testing is only available at specialized laboratories as a diagnostic service. Bartter syndrome treatment requires lifelong administration of certain supplements and medications. Presently, there is no cure for Bartter syndrome; however, restoring the proper balance of fluids and electrolytes in the body with certain medication can effectively work as Bartter syndrome treatment. For more Insights into the Market, Request a Sample of this Report What is Driving the Market? The growing prevalence of kidney disorders and increasingly unhealthy lifestyle are the major factors driving the growth of the global Bartter syndrome treatment market. Moreover, the rising number of clinical trials is expected to drive the Bartter syndrome treatment market. Strong R&D and increasing awareness in low-middle income countries are further responsible for driving the growth of the global Bartter syndrome treatment market over the forecast period. Generally, Bartter syndrome affects both males and females in equal numbers. According to NORD, Bartter syndrome affects around one in 1,000,000 individuals in the overall population. Request Detailed Table of Content Here: Which Segment Dominates Global Market? Geographically, the global Bartter syndrome treatment market has been segmented into North America, Latin America, Europe, CIS & Russia, Asia Pacific excluding Japan (APEJ), Japan, and the Middle East and Africa (MEA). North America is anticipated to be the dominant market in the global Bartter syndrome treatment market, owing to the increasing prevalence of kidney disorders. Europe is expected to be the second-largest market in the global Bartter syndrome treatment market throughout the forecast period, owing to high awareness regarding health-related problems in the region. The Bartter syndrome treatment market in the Asia Pacific region is expected to grow with the maximum CAGR, mostly due to the growing geriatric population and increasing number of healthcare facilities in the region. Examples of some of the key players operating in the global Bartter syndrome treatment market include Abbott Laboratories, Novartis AG, Pfizer Inc., Eli Lilly and Company, Bristol-Myers Squibb Company and others. Key players operating in the global Bartter syndrome treatment market are majorly focusing on product launches through strategies such as mergers and acquisitions. 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