Improved Guidelines for Patient Advocacy Groups Supporting Rare Diseases and Working with Pharmaceutical Companies

A study appeared recently in the Orphanet Journal of Rare Diseases reporting that rare diseases affect 350 million people worldwide.  The definition of rare disease differs between the United States…

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An Experimental Treatment for POMC and LEPR Deficiency Obesity Appears Effective

According to a story from sectorpublishingintelligence.co.uk, the biopharmaceutical company Rhythm Pharmaceuticals, Inc., has recently announced the results from two phase 3 clinical trials. These trials were testing the company's experimental…

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FDA Permits Retrotrope’s Phase 2/3 Study of Infantile Neuroaxonal Dystrophy Drug Candidate
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FDA Permits Retrotrope’s Phase 2/3 Study of Infantile Neuroaxonal Dystrophy Drug Candidate

According to a press release from the California-based biotechnology company Retrotrope, Inc., the Company has completed recruitment for its upcoming phase 2/3 study of RT001, an experimental drug designed for…

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Editing Gut Bacteria Was Able to Curb Colorectal Cancer in Mice
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Editing Gut Bacteria Was Able to Curb Colorectal Cancer in Mice

According to a story from EurekAlert!, a team of researchers affiliated with UT Southwestern were successfully able to curtail inflammation-associated colorectal cancer in mice by precisely editing the animals' gut…

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A Potential Treatment for Familial Chylomicronemia Syndrome Performs Well in Trials

According to a story from EurekAlert!, the results of a recent clinical trial should get the attention of patients in the US with the rare disorder familial chylomicronemia syndrome. The…

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Large Scale Trial Aims to Test Precision Medicines Based on Genomic Profiling in a Variety of Cancers
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Large Scale Trial Aims to Test Precision Medicines Based on Genomic Profiling in a Variety of Cancers

According to a story from Newswise, Quest Diagnostics, a laboratory that specializes in diagnostic information, has announced plans to participate in a unique, large scale trial called NCI-MATCH, which stands…

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