PTC Therapeutics announced that the US Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) regarding the company’s New Drug Application (NDA) for vatiquinone, an investigational therapy intended…
According to a recent news release from PR Newswire, the European Medicines Agency (EMA) has recently awarded elamipretide its Orphan Drug designation for the treatment of Barth syndrome. This treatment,…
Understanding a rare disease is the first step in treating it. How can you combat the underlying mechanisms of a disorder if you do not know what they are? A…
To determine disease progression and severity, researchers often use biomarkers, a measurable substance that indicates disease state. According to Myasthenia Gravis News, a new study shows that acetylcholine receptor (AChR)…
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