With Its Own Disease Code, Angelman Syndrome Will be Easier to Research and Monitor
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With Its Own Disease Code, Angelman Syndrome Will be Easier to Research and Monitor

According to a story from Angelman Syndrome news, Angelman syndrome was recently given its own disease unique disease code: Q93.51. The new code will help make researching and tracking the…

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Angelman Syndrome Treatment Being Developed is Awarded ‘Orphan Drug’ Status in the U.S.
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Angelman Syndrome Treatment Being Developed is Awarded ‘Orphan Drug’ Status in the U.S.

A new drug being developed to treat Angelman syndrome (AS) has just been approved for ‘orphan drug’ status in the U.S., reports GlobeNewswire. The drug, called GTX-101, works by activating the…

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An Experimental Treatment for Fragile X and Angelman Syndromes Gets Fast Track Designation
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An Experimental Treatment for Fragile X and Angelman Syndromes Gets Fast Track Designation

According to a story from pharmiweb.com, the pharmaceutical company Ovid Therapeutics announced recently that the U.S. Food and Drug Administration (FDA) has given its drug candidate OV101 Fast Track designation…

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The FDA is Beginning to Accept New Endpoints and “Real World” Data in Clinical Trials
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The FDA is Beginning to Accept New Endpoints and “Real World” Data in Clinical Trials

According to a story from Charcot-Marie-Tooth Disease News, the U.S. Food and Drug Administration (FDA), under the leadership of commissioner Scott Gottlieb, has begun to change clinical trials by accepting…

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Tax Bill Threatens Continued Therapy and Drug Advancement for Rare Diseases
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Tax Bill Threatens Continued Therapy and Drug Advancement for Rare Diseases

Originally reported by Fox 17 Nashville, many Tennessee residents stress that a new tax reform bill will eliminate continued drug advancement and development for many rare diseases. The House and…

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Photographer Captures Daily Life of Rare Families with Dignity and Grace
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Photographer Captures Daily Life of Rare Families with Dignity and Grace

Rare disease began shaping acclaimed photographer, Karen Haberberg's family years before she was born. Her parents had given birth to her older brother, who was diagnosed with Tay-Sachs. Tay-Sachs is a…

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