14th ICIEM Conferences: Data to be Presented on Fabry Disease and Gaucher Disease Trials
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14th ICIEM Conferences: Data to be Presented on Fabry Disease and Gaucher Disease Trials

From November 21st until the 23rd, medical professionals and others involved in the metabolic field will come together for the 14th International Congress of Inborn Errors of Metabolism (ICIEM). It…

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11/13: Indiana Blood Drive Supports Girl with Opsoclonus-Myoclonus Syndrome
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11/13: Indiana Blood Drive Supports Girl with Opsoclonus-Myoclonus Syndrome

3-year-old Ruby Ann Grimes is bubbly, happy, and loving. But each and every day, Ruby faces an ongoing battle: Opsoclonus-Myoclonus syndrome (OMS). Last January (2020), Ruby was first diagnosed with…

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Study Shows Novel BTK Degrader Has Meaningful Results for Patients with Chronic Lymphocytic Leukemia

According to a recent article, a study looking at an investigational BTK degrader proved to have important results in patients with chronic lymphocytic leukemia. Chronic Lymphocytic Leukemia (CLL) Chronic lymphocytic…

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FDA Grants Rare Pediatric Disease Designation to AVR-RD-05 for Hunter Syndrome
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FDA Grants Rare Pediatric Disease Designation to AVR-RD-05 for Hunter Syndrome

According to an article published in Yahoo, the FDA has recently granted the Rare Pediatric Disease designation to AVR-RD-05, a gene therapy for Hunter syndrome. This designation is reserved for…

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How Lina Williamson Works to Empower the Ehlers-Danlos Syndrome Community
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How Lina Williamson Works to Empower the Ehlers-Danlos Syndrome Community

“The patient network is incredibly important,” Lina Williamson, PhD, explained to me. You see, in her childhood, Lina experienced a number of seemingly unexplainable symptoms: severe and sometimes debilitating leg…

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New Collaboration Undertakes CANDID Study for CDKL5
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New Collaboration Undertakes CANDID Study for CDKL5

In a late October news release from pharmaceutical company Marinus Pharmaceuticals, Inc. ("Marinus"), the company shared that it would be collaborating with the Loulou Foundation, a private UK nonprofit dedicated…

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