ICYMI: More Steps Required for Experimental ADPKD Drug in Order to Obtain Orphan Designation
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ICYMI: More Steps Required for Experimental ADPKD Drug in Order to Obtain Orphan Designation

According to a story from thenewsreports.com, the biopharmaceutical company XORTX Therapeutics, Inc., recently announced that the company and its partner Cato Clinical Research have received correspondence from the US Food…

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New Algorithm May Diagnose Cystic Fibrosis Faster than Physicians
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New Algorithm May Diagnose Cystic Fibrosis Faster than Physicians

A diagnosis means everything for a rare disease patient. However, to process 200 patient records manually, it would take the most dedicated physician approximately 40 hours. That's one full work…

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ICYMI: Experimental Treatment for Duchenne Muscular Dystrophy Shows Potential in Early Trial
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ICYMI: Experimental Treatment for Duchenne Muscular Dystrophy Shows Potential in Early Trial

According to a story from globenewswire.com, the biotechnology company Wave Life Sciences Ltd. recently announced that the tolerability and safety data from its Phase 1 clinical trial of its investigational therapy…

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Nplate Now Approved for Pediatric Patients with Immune Thrombocytopenia!
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Nplate Now Approved for Pediatric Patients with Immune Thrombocytopenia!

Immune Thrombocytopenia (ITP) is diagnosed by low platelet counts in the blood. It also causes impaired platelet production. This put patients at risk for serious bleeding as well as spontaneous bruising.…

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Potential Gene Therapies for Tay-Sachs, Sandhoff, and GM1 Gangliosidosis Now Have Worldwide Licenses
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Potential Gene Therapies for Tay-Sachs, Sandhoff, and GM1 Gangliosidosis Now Have Worldwide Licenses

Tay-Sachs disease and Sandhoff disease (also known as GM2 Gangliosidosis), and GM1 Gangliosidosis are neurodegenerative conditions. They are a result of a defective enzyme. This enzyme causes toxic gangliosides to accumulate within the…

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New Orphan Drug Designation for Potential Spinocerebellar Ataxia Treatment
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New Orphan Drug Designation for Potential Spinocerebellar Ataxia Treatment

Spinocerebellar Ataxia (SCA) refers to a group of neurodegenerative disorders. Ataxias are a component of the nervous system which control movement. SCA occurs when the ataxias in the brain or spinal…

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This Just in: Data from Phase 3 Trial for Familial Adenomatous Polyposis is Coming Early 2019
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This Just in: Data from Phase 3 Trial for Familial Adenomatous Polyposis is Coming Early 2019

Familial adenomatous polyposis (FAP) is a condition which causes extra tissue to form in various parts of the body such as the large intestine and gastrointestinal track. FAP affects about…

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