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Genetic and Rare Diseases Information Center (GARD)

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Congenital generalized lipodystrophy type 4

Other Names for this Disease
  • Berardinelli-Seip congenital lipodystrophy, type 4, with muscular dystrophy
  • CGL4
  • GCL4
  • Generalized congenital lipodystrophy with myopathy
  • Lipodystrophy, Berardinelli-Seip congenital, type 4, with muscular dystrophy
More Names
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Clinical Trials & Research for this Disease

  • Orphanet lists clinical trials, research studies, and patient registries enrolling people with this condition. Click on Orphanet to view the list.

Medical Products

The medication(s) listed in the table(s) below have been approved by the Food and Drug Administration (FDA) for treatment of this condition. The FDA Office of Orphan Products Development designates "orphan products" for those that treat rare diseases affecting fewer than 200,000 Americans. The table(s) below may not be an exhaustive list of drugs or products used to treat this condition. There may be other products available that are not considered orphan products. To search for all FDA approved drugs, visit Drugs@FDA. You can find orphan products used to treat other conditions by searching the Orphan Drug Product Designation database.

Generic Name metreleptin
Trade Name
(Manufacturer Name)
(Amylin Pharmaceuticals, LLC)
The FDA has approved this product to be used in this manner.
Adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy.
More Information about this product Drug Information Portal