Rare Diseases

Rare Diseases Perspective


Rare Diseases News & Journal Articles

  • Alexion's Rare Blood Disorder Drug Succeeds in Late-stage Study Alexion Pharmaceuticals Inc said on Thursday its experimental drug to treat patients with the rare blood disorder, paroxysmal nocturnal hemoglobinuria (PNH), was shown to be not inferior to its flagship drug, Soliris, which could help the company maintain its lock on that market as competition looms.
  • Exercising to Death: The Rare Risk of Rhabdo Rhabdomyolysis is a syndrome involving muscle breakdown and damage. When muscles are injured, they release their contents, including a muscle enzyme, into the bloodstream. The disorder is dangerous but rare. In one study, 22 people out of 100,000 were known to have it.

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