Results From the Longest Familial Hypercholesterolemia Study to be Announced at the ACC 2019 Conference

New Research to be Presented In just a few days the American College of Cardiology will host their 68th Annual Scientific Session (ACC.19). It will be held in New Orleans,…

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Simple Blood Test Could Prevent Serious Side Effects for Inflammatory Bowel Disease Treatment

Inflammatory Bowel Disease Inflammatory bowel disease (IBD) is the collective term used to describe ulcerative colitis and Crohn's disease. These conditions cause diarrhea, rectal bleeding, fatigue, weight loss, and rectal bleeding. These…

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The Argument for Mandatory Newborn Screening and Government Subsidies for Rare Diseases in India

The State of Rare Disease in India There are 7,000 known rare diseases worldwide. The Organization for Rare Diseases India (ORDI) estimates 70 million people are affected by a rare diagnosis…

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New Potential Treatment for Systemic-Onset Juvenile Idiopathic Arthritis Investigated in China

SOJIA SOJIA stands for systemic-onset Juvenile Idiopathic Arthritis (JIV). It is a very severe form of JIA which causes joint pain, inflammation, recurrent fevers, and a skin rash. Patients often take predinsolone,…

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4,800 People Waited for Hours in the Rain to See if They Were Eligible Stem Cell Donors for Little Boy with Rare Leukemia

On December 28, 2018, five-year-old Oscar Saxelby-Lee was diagnosed with a rare and aggressive form of leukemia. Now he's in a race against time to find an eligible stem cell…

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Investigational Drug for Charcot-Marie-Tooth Disease Earns Orphan Drug Designation
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Investigational Drug for Charcot-Marie-Tooth Disease Earns Orphan Drug Designation

According to a story from Central Charts, the biopharmaceutical company Acceleron Pharma Inc. recently announced that the company's experimental drug candidate ACE-083 has earned Orphan Drug designation from the US…

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Study Testing Gene Therapy for Charcot-Marie-Tooth Disease Earns Funding from MDA and CMTA

According to a story from The Muscular Dystrophy Association (MDA), the organization alongside the Charot-Marie-Tooth Association (CMTA) has awarded grant funding totaling $276,430 to a research study that will develop…

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