Rare Diseases and Orphan Drugs

The X-linked disorders defined as the males with the disorder are the more affected than the females and in the same case they may be lethal during the development after birth so the effected females can survive. The X-linked disease is reported in the large pedigree and the disease is characterized by a dilated neutropenia and skeletal myopathy . and the untreated patients all boys are died in infancy and the childhood from cardiac decomposition.

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