New Drug Approved in the EU for Treating Hereditary Transthyretin Amyloidosis
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New Drug Approved in the EU for Treating Hereditary Transthyretin Amyloidosis

According to a story from bioportfolio.com, two companies, Ionis Pharmaceuticals, Inc., and its affiliate Akcea Pharmaceuticals, recently announced that its product inotersen (to be marketed as TEGSEDI) has gained approval…

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An Experimental Drug For Treating Duchenne Muscular Dystrophy Will be Studied in a Phase 3 Trial, Catabasis Says
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An Experimental Drug For Treating Duchenne Muscular Dystrophy Will be Studied in a Phase 3 Trial, Catabasis Says

Catabasis Pharmaceuticals Inc. has announced plans for a Phase 3 clinical trial called Polaris DMD. It will investigate the experimental drug edasalonexent for the treatment of patients with Duchenne muscular…

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How Rare is Eosinophilic Esophagitis Anyway? A Family’s Story Brings Awareness
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How Rare is Eosinophilic Esophagitis Anyway? A Family’s Story Brings Awareness

According to a story from the Washington Times-Reporter, Michelle Marcinak's three sons-- John Henry, Leo, and Rafe-- were all diagnosed with the rare gastric condition eosinophilic esophagitis. Early symptoms of…

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Investigational Drug for Facioscapulohumeral Muscular Dystrophy Gets Orphan Drug Designation
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Investigational Drug for Facioscapulohumeral Muscular Dystrophy Gets Orphan Drug Designation

According to a story from BioSpace, the biopharmaceutical company Acceleron Pharma, which develops TGF-beta based treatments for rare and serious illnesses, recently announced that the U.S. Food and Drug Administration…

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Kids With Stevens-Johnson Syndrome and Toxic Epidermal Necrolysis Experience Complications and Get Inconsistent Treatment, Study Says
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Kids With Stevens-Johnson Syndrome and Toxic Epidermal Necrolysis Experience Complications and Get Inconsistent Treatment, Study Says

According to a story from ScienceDaily, a recent analysis has been conducted in order to investigate patient outcomes for children with the rare skin condition Stevens-Johnson syndrome, as well its…

Continue Reading Kids With Stevens-Johnson Syndrome and Toxic Epidermal Necrolysis Experience Complications and Get Inconsistent Treatment, Study Says