Experimental Treatment for Biliary Atresia Gets Orphan Drug Designation in the EU
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Experimental Treatment for Biliary Atresia Gets Orphan Drug Designation in the EU

According to a story from Market Screener, the drug developer Albireo Pharma, Inc., recently announced that its investigational product candidate A4250 has earned Orphan Drug designation from the European Commission.…

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Device Allows Monitoring of Lung Function at Home For Duchenne Muscular Dystrophy Patients
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Device Allows Monitoring of Lung Function at Home For Duchenne Muscular Dystrophy Patients

According to a story from EurekAlert!, monitoring lung function is an important role of managing Duchenne muscular dystrophy and other diseases that can cause wasting of the respiratory muscles. Many…

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New Phase 3 Trial for Prader-Willi Syndrome is now Enrolling Patients
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New Phase 3 Trial for Prader-Willi Syndrome is now Enrolling Patients

Prader-Willi syndrome (PWS) is a neurodevelopmental disorder which causes intellectual disability, a deficiency of growth hormones, compulsive behavior, a high risk of obesity, and hyperphagia, a symptom which can be…

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New Mutations Identified in Study of Five Polish Families With Choroideremia

According to a scientific study published in the Orphanet Journal of Rare Diseases, a team of researchers were able to identify the mutations responsible for choroideremia in five families of Polish descent.…

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