Roelofs et al., "Adipose specific disruption of seipin causes early-onset
generalised lipodystrophy and altered fuel utilisation without severe metabolic disease," Molecular Metabolism, vol.
Echocardiographic assessment of cardiac function and morphology in patients with
generalised lipodystrophy. Eur J Pediatr 1985;144:355-359.
The product is an orphan drug product to treat complications of leptin deficiency in patients with
generalised lipodystrophy. It is a recombinant human leptin analogue indicated as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired
generalised lipodystrophy.
The causal gene 'AG-PAT2' which is involved in the synthesis of phosphatidic acid and triglycerides, was removed in the mice--resulting in rodents with
generalised lipodystrophy.
The causal gene 'AGPAT2' which is involved in the synthesis of phosphatidic acid and triglycerides, was removed in the mice-resulting in rodents with
generalised lipodystrophy.
The firm stated that a version of Leptin has recently been approved in the United States for treating
generalised lipodystrophy and also in Japan.
Medicine company AstraZeneca (NYSE:AZN) revealed on Tuesday the receipt of approval from the US Food and Drug Administration (FDA) for orphan drug Myalept for treating leptin deficiency in patients with congenital or acquired generalised lipodystrophy.
The company added that Myalept (metreleptin for injection) is a recombinant human leptin analog indicated as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalised lipodystrophy.