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Rare Diseases

This ASHP Resource Center is an area for pharmacists to obtain additional information and resources about rare disease and related topics. This resource center is managed by the Section of Clinical Specialists and Scientists.
The Management of Patients with Rare Diseases Resource Center contains a compilation of resources that pharmacy professionals may find helpful for … 
  • Developing their understanding of rare disorders (any disease, disorder, illness or condition affecting fewer than 200K people in the US is considered rare)

  • Expanding their knowledge base of rare disease resources (common disease states and resources are well known, resources related to rare diseases are less known)

  • Keeping up-to-date on new drug developments and orphan drug products within the specialty (more than 90% of rare diseases are without an FDA-approved treatment)

These resources are to be utilized to help guide pharmacy practice in the management of patients with rare disorders.

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