Emphysematous hepatitis with successful treatments: A rare case report
Sign Up to like & getrecommendations! Published in 2023 at "Medicine"
DOI: 10.1097/md.0000000000032530
Abstract: Rationale: Emphysematous hepatitis (EH) is a rare and fulminant gas-forming liver infection. Only 3 patients were successfully treated. Diabetes mellitus and a history of digestive system cancer may predispose individuals to EH. Computed tomography (CT)… read more here.
Keywords: treatments rare; hepatitis successful; case; hepatitis ... See more keywords
Patient organization involvement and the challenge of securing access to treatments for rare diseases: report of a policy engagement workshop
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DOI: 10.1186/s40900-017-0065-z
Abstract: Plain English summaryPatients with rare diseases often help to develop new treatments for their conditions. But once developed, those treatments are sometimes priced too high for many patients to access them. We became aware that… read more here.
Keywords: policy; challenge; rare diseases; treatments rare ... See more keywords
Development and validation of targeted treatments for the rare tumor syndrome infantile myofibromatosis.
Sign Up to like & getrecommendations! Published in 2020 at "Journal of Clinical Oncology"
DOI: 10.1200/jco.2020.38.15_suppl.e22519
Abstract: e22519Background: Infantile myofibromatosis (IMF; MIM 228550) is an autosomal dominant disorder characterized by fibroblastic tumors of skin, muscles, viscera and bones. Tumor growth may begin duri... read more here.
Keywords: validation targeted; development validation; infantile myofibromatosis; treatments rare ... See more keywords