Articles: disease.
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Long-chain fatty acid oxidation disorders (LC-FAODs) represent a group of rare inborn errors of metabolism characterized by acute crises of energy metabolism and severe energy deficiency. Clinical manifestations include rhabdomyolysis, liver dysfunction, severe hypoglycemia, and cardiomyopathy. The symptoms associated with these genetic disorders may result in frequent hospitalizations and early death. ⋯ Triheptanoin was recently approved for the treatment of LC-FAODs. As with any novel agent for rare diseases, the treatment adoption and coverage criteria for rare disease therapy such as this presents challenges for managed care decision makers. Both managed care decision makers and specialty pharmacists must ensure that healthcare professionals provide appropriate patient and caregiver education surrounding the management of LC-FAODs, including vital information on adopting emerging agents into the therapy armamentarium for these disorders.
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Social distancing, implemented to decrease the spread of coronavirus disease-2019 (COVID-19), forced major changes in medical practices, including an abrupt transition from face-to-face to remote patient care. Pre-clinical medical studies were concomitantly switched to electronic distance learning. ⋯ The high satisfaction and the positive experience with the electronic distance learning imposed by the COVID-19 epidemic implied a successful transition and might induce future changes in pre-clinical medical studies.
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Revista médica de Chile · Aug 2020
Case Reports[DRESS syndrome induced by sulfasalazine. Report of one case].
DRESS syndrome (Drug Reaction with Eosinophilia and Systemic Symptoms) is a severe, rare and potentially lethal idiosyncratic condition associated with the use of some drugs. Given its broad spectrum of presentation, clinical suspicion is essential for management, since it requires the immediate withdrawal of the culprit drug, support measures and the use of corticosteroids as the first line of treatment. We report a 24-year-old woman with a diagnosis of ulcerative colitis with joint involvement despite the use of infliximab, who presented symptoms, signs and laboratory compatible with DRESS syndrome on the third week after indicating sulfasalazine for her baseline disease.
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In the coronavirus disease 2019 (COVID-19) era, clinical registries and innovative virtual care delivery tools should be leveraged to engage populations in effective chronic disease management.