Lipodystrophy in the news

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Health Canada has approved Chiesi Global Rare Diseases' Myalept as an adjunct to diet for treating complications of leptin deficiency in lipodystrophy. The approval covers patients with congenital or acquired generalized lipodystrophy and familial partial lipodystrophy who haven't responded to standard treatments. Myalept, developed by Amryt Pharma and acquired by Chiesi Group, showed significant efficacy in reducing metabolic abnormalities in both generalized and partial lipodystrophy patients, with common adverse reactions including weight loss, hypoglycemia, and fatigue.

Through mouse models, researchers discovered that enhancing NRF2 expression, a transcription factor regulating various cellular pathways including oxidative stress response, in the liver can protect against lipodystrophy-induced hepatic damage. Their research, recently published in the International Journal of Molecular Sciences, demonstrates that liver-directed NRF2 targeting could offer therapeutic opportunities to mitigate the effects of lipodystrophy, potentially preventing fatty liver disease and other metabolic complications associated with the condition.