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GT Biopharma Begins Trial for Treatment of Rare Muscular Disease
GT Biopharma announced it started the proof of concept phase 1 clinical trial with GTP-004 against rare muscular disease myasthenia gravis.
GT Biopharma (OTCQB:GTBP) announced it started the proof of concept phase 1 clinical trial with GTP-004 against rare muscular diseasemyasthenia gravis.
As quoted in the press release:
The prevalence of myasthenia gravis in the United States is estimated at 14 to 20 per 100,000 population, approximately 36,000 to 60,000 cases in the U.S. (Howard, 2015). The hallmark of the disease is muscle weakness that increases during periods of activity and improves after periods of rest. Muscular weakness can be generalized or localized to certain muscle groups, and involvement of the bulbar and respiratory muscles can be life-threatening (Phillips and Vincent, 2016). The disease occurs in all ethnic groups and both genders. Onset commonly occurs in young adult women (under 40 years) and older men (over 60 years), but it can occur at any age (NINDS, 2017). Rarely, children may show signs of congenital myasthenia or congenital myasthenic syndrome (CMS). These are not autoimmune disorders but are caused by defective genes that produce abnormal proteins instead of those that normally are involved in the cholinergic transmission.
The treatment of myasthenia gravis involves treatment of the muscular weakness by acetylcholinesterase inhibitors that do not cross the blood-brain barrier and by immunotherapy to slow disease progression (Gotterer and Li, 2016).
Click here to read the full press release.
Source: www.accesswire.com
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