Leptin replacement therapy with human recombinant leptin metreleptin has been shown to be an effective therapy to alleviate the metabolic complications associated with<unk> , and has been approved by the FDA for the treatment of generalized<unk>  syndromes. In Europe based on EMA, metreleptin should be used in addition to diet to treat<unk> , where patients have loss of fatty tissue under the skin and build-up of fat elsewhere in the body such as in the liver and muscles. The medicine is used in: adults and children above the age of two years with generalised<unk>  (Berardinelli-Seip syndrome and Lawrence syndrome) and in adults and children above the age of 12 years with partial<unk>  (including Barraquer-Simons syndrome), when standard treatments have failed. Volanesorsen is an Apo-CIII inhibitor that is currently being investigated as a potential therapeutic to reduce hypertriglycerides in Familial Partial<unk>  patients in the BROADEN study.
Lipodystrophy
Lymphedema
Osteodystrophy
Scarring hair loss
Acanthosis nigricans
Presbyphagia
Pseudodysphagia
Trichomegaly