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Engineers from the Engineering Faculty at the University of Sheffield and Boston Children’s Hospital, Harvard Medical School have created a robot with the intention of esophageal atresia .
An orphan drug designation request for Vitality Biopharma’s prodrug VITA-100, a cannabinoid that treats pediatric ulcerative colitis, has been sent to the U.S. FDA.
The FDA has accepted for review supplemental Biologics License Application for Ruconest for routine prophylaxis to prevent attacks in adult and adolescent patients with hereditary angioedema, Pharming Group N.V. reported this morning.
Researchers at St. Jude Children’s Research Hospital have developed a new gene therapy that creates fully-functioning immune systems in babies diagnosed with severe combined immunodeficiency, commonly referred to as the “Bubble Boy” disease.
Results from a study report that behavioral interventions, like environmental enrichment, can reduce the functional deficit experienced by patients with Rett syndrome.
Results from a study report that behavioral interventions, like environmental enrichment, can reduce the functional deficit experienced by patients with Rett syndrome.
After discovering the fate of a former teammate’s child, wrestlers at Wells High School in Maine are using their sport as an incentive to raise money for Sanfilippo Syndrome.
 
Data published in the Orphanet Journal of Rare Diseases suggests that endurance training could have a positive impact in Huntington’s disease patients, much like it has in anyone else.
 
Data published in the Orphanet Journal of Rare Diseases suggests that endurance training could have a positive impact in Huntington’s disease patients, much like it has in anyone else.
 
Adamas Pharmaceuticals, Inc. announced the full commercial launch of Gocovri (amantadine) extended release capsules for the treatment of dyskinesia in patients with Parkinson’s disease receiving levodopa-based therapy, with or without concomitant dopaminergic medications.
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