Company Plans Extension Study for Phase 3 Trial of Amyotrophic Lateral Sclerosis Drug
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Company Plans Extension Study for Phase 3 Trial of Amyotrophic Lateral Sclerosis Drug

According to a story from pm360online.com, the Orion Corporation, a pharmaceutical company based in Finland, has recently announced that it plans to continue its Phase 3 trial of levosimendan as…

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Company Earns Orphan Drug Designation for Autoimmune Hepatitis From the FDA
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Company Earns Orphan Drug Designation for Autoimmune Hepatitis From the FDA

According to a story from finanznachrichten.de, the Swedish drug company Calliditas Therapeutics has recently announced that the US Food and Drug Administration (FDA) has given one of the company's experimental drugs…

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Potential Gene Therapy for Late Infantile Batten Disease Earns Rare Pediatric Disease Designation

According to a story from BioSpace, the biotechnology company REGENXBIO, Inc. recently announced that its experimental gene therapy candidate called RGX-181 has been granted Rare Pediatric Disease designation from the…

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Study Shows Treatment may be Effective for Chronic Kidney Disease but Increase Risk of Hyperkalemia

A recent study, called TOPCAT, evaluated the effects of spironolactone as a treatment for individuals living with Chronic Kidney Disease (CKD) who are also diagnosed with heart failure with preserved ejection fraction,…

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Importance of Patient-Centered Research in Familial Chylomicronemia Syndrome
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Importance of Patient-Centered Research in Familial Chylomicronemia Syndrome

Familial Chylomicronemia Syndrome (FCS) Is a rare disease caused by malfunctioning lipoprotein lipase. This results in a buildup of triglycerides in the body's plasma. FCS can cause pancreatitis, memory loss, nerve…

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Possible Treatment for Niemann-Pick Disease Type C Performs Well in Clinical Trial
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Possible Treatment for Niemann-Pick Disease Type C Performs Well in Clinical Trial

According to a story from labiotech.eu, the biotechnology company Orphazyme has been developing an experimental therapy for Niemann-Pick disease type C. In a recent Phase 2/3 clinical trial, the investigational…

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