Genzyme Corporation

Henry Hoppe, Iv

Seng H Cheng

Alan E Smith

Shaona Lee Fang

Methods for treating cystic fibrosis are described. The methods involve the administration of a protein enhancing agent, differentiating agent and/or carboxy-compound to a subject afflicted with cystic fibrosis such that mutant cystic fibrosis transmembrane regulator protein present within cystic fibrosis-associated cells becomes functional. Other aspects described include therapeutic compositions and packaged drugs.

Methods and therapeutic compositions for treating cystic fibrosis

Methods and compositions for treating Cystic Fibrosis by mobilizing mutant forms of CFTR, which retain at least some functional activity, to the plasma membrane where they can mediate chloride ion transport are disclosed.

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Acton, MA, United States of America

Henry Hoppe, Iv