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Treating Genetic Diseases

  Sample User Question Comments/Analysis Step By Step Guide Additional Tips  

Progress is being made in developing drugs to treat rare and gentic diseases.

Sample User Question back to top

 
You briefly spoke with a colleague in the hospital corridor who told you about a new drug treatment for X-linked juvenile retinoschisis (XLRS). You did not write down the name of the drug. Where would you find it?
 

Comments / Analysis back to top

The FDA website provides an efficient means to search for new drugs as they come on the market.

Step By Step Guide back to top

  1. FDA Cumulative List of Orphan Products Designated Year to Date - From the FDA's Orphan Drug home page, click on the Designating an Orphan Product: Drugs and Biologics link.
  2. From the Orphan Product Designation Page click on the Lists of Orphan Product Designations and Approvals link.
  3. Next, review the List of all Orphan Drug Designated Products that have received Market Approval, year-to-date.


    • X-linked juvenile retinoschisis
      • Tip: If the drug were not located in the Year to Date list, check the Cumulative List of all products that have received Orphan Drug Designation.


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