Phase 2b Prader-Willi Syndrome Trial Ends in Disappointment
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Phase 2b Prader-Willi Syndrome Trial Ends in Disappointment

According to a story from Malvern Daily Record, the biopharmaceutical company Millendo Therapeutics, Inc. recently released data from its phase 2b clinical trial. This study was testing the company's experimental…

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Experimental Non-Steroid Treatment Displays Potential for Congenital Adrenal Hyperplasia
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Experimental Non-Steroid Treatment Displays Potential for Congenital Adrenal Hyperplasia

According to a story from Healio, the results from a recent phase 2A clinical trial have demonstrated the effectiveness of an experimental drug in treating congenital adrenal hyperplasia (CAH). The…

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This Young Man Accurately Diagnosed His Own Disorder But Doctors Did Not Believe His Diagnosis

Doug Lindsay ran track in high school. According to an article published by CNN Health, Doug had big plans as a senior at Rockhurst University in Kansas City where he…

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An Addison’s Disease Story: Multiple Rare Diagnoses Often Means a Longer Waiting Game

Emily Dover  This is Emily Dover's story. At birth- perfectly healthy newborn, smaller than her siblings (8 pounds) 1 week- was having trouble sleeping and began crying in pain 4…

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Proof of Concept Results Achieved in Congenital Adrenal Hyperplasia Trial

According to a story from BioSpace, the biotechnology company Spruce Biosciences recently announced that they have achieved positive proof of concept data in the company's clinical trial. This is a…

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The FDA has Awarded Over $18 Million for Rare Disease Research
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The FDA has Awarded Over $18 Million for Rare Disease Research

The United States Food and Drug Administration has awarded twelve research grants worth a total of over $18 million for research into rare diseases. For more information about this news,…

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Chin Hairs in Women Could be Indicators of Cushing Syndrome or Congenital Adrenal Hyperplasia
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Chin Hairs in Women Could be Indicators of Cushing Syndrome or Congenital Adrenal Hyperplasia

According to a story from msn.com, a significant portion of woman may notice a small number of hairs growing on their chin at some point in their lives. In most…

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This Experimental Drug Just Received Orphan Drug Designation For Congenital Adrenal Hyperplasia
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This Experimental Drug Just Received Orphan Drug Designation For Congenital Adrenal Hyperplasia

According to a story from prnewswire.com, the experimental treatment SPR001 was just granted orphan drug status by the Food and Drug Administration (FDA). The new treatment is currently being developed…

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