Rare Diseases

Rare Diseases Perspective


Rare Diseases News & Journal Articles

  • Alnylam's RNAi Drug Succeeds in Key Study, Shares Soar Alnylam Pharmaceuticals Inc's RNAi-based drug that targets a rare genetic disease met the main goal of a key study, in a breakthrough for the new class of medicines that works by blocking pathogenic proteins.
  • Alert Misdosing Ocaliva for Liver Disorder Can Be Fatal, FDA Says Some patients with primary biliary cholangitis have developed serious liver injury or have died after receiving higher doses of obeticholic acid than the FDA recommends in the product label.

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