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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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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Online Community Helped Free a Family Affected by Coffin-Siris Syndrome From Isolation

According to a story from the Globe and Mail, Michelle Quinlan was overwhelmed when her child Avery was diagnosed with Coffin-Siris syndrome, a rare genetic disease that can cause intellectual…

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Five Benefits of the New Pig Models of Huntington’s Disease, According to Scientists
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Five Benefits of the New Pig Models of Huntington’s Disease, According to Scientists

New pig models of Huntington’s disease have been developed, and they are expected to have several benefits compared to existing rodent models of the condition. Read the source article here…

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Kalytera Therapeutics Will Meet with the FDA to Discuss Cannabidiol as a Possible Treatment For Acute GVHD
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Kalytera Therapeutics Will Meet with the FDA to Discuss Cannabidiol as a Possible Treatment For Acute GVHD

Kalytera Therapeutics, Inc. has announced that a meeting with the Centre for Drug Evaluation and Research of the US FDA is planned for Tuesday, 31st July this year. The purpose of…

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Could Virtual Reality Cure Glaucoma?
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Could Virtual Reality Cure Glaucoma?

According to a story published by STAT, a daredevil neuroscientist at Stanford University is utilizing the relatively new concept of virtual reality to solve an unsuspecting problem. Not only is…

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BrainStorm Has Secured a Second Manufacturing Facility for NurOwn, an Experimental Drug for ALS

BrainStorm Cell Therapeutics Inc. has announced that it has secured its second U.S. manufacturing location for NurOwn®, an experimental drug currently under investigation for the treatment of amyotrophic lateral sclerosis…

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First Hemophilia B Patient Enrolled in Potential Breakthrough Phase III Trial

According to BioPortfolio, the company UniQure has recently announced that the first hemophilia B patient has been enrolled in their Phase III study of the treatment AMT-061, which is an…

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