Protein Replacement Therapies for Rare Diseases: A Breeze for Regulatory Approval?

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  Protein Replacement Therapies for Rare Diseases: A Breeze for Regulatory Approval?

  By Jennifer A. Gorzelany, Mark P. de Souza

  Science Translational Medicine27 Mar 2013 : 178fs10

  Protein replacement therapies for rare monogenic diseases have a higher probability of regulatory approval compared with biologics, small molecules, and grant-funded orphan drugs.

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  Protein Replacement Therapies for Rare Diseases: A Breeze for Regulatory Approval?

  By Jennifer A. Gorzelany, Mark P. de Souza

  Science Translational Medicine27 Mar 2013 : 178fs10

  Protein replacement therapies for rare monogenic diseases have a higher probability of regulatory approval compared with biologics, small molecules, and grant-funded orphan drugs.

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