title: Gaucher disease, enzyme replacement therapy, and the Patient Assistance Program.
abstract: Gaucher disease is the most common lysosomal storage disorder characterized by a missing enzyme, glucocerebrosidase. Enzyme replacement therapy has been available in the form of Ceredase (Genzyme Corporation, Cambridge, Massachusetts) (alglucerase injection) since 1991, and in the form Cerezyme (Genzyme Corporation, Cambridge, Massachusetts) (imiglucerase for injection) since 1994. Because of the high cost associated with enzyme replacement therapy, the Genzyme Corporation, manufacturers of Ceredase and Cerezyme, developed the Patient Assistance Program. The program assisted those patients who chose to use it with the facilitation of coverage approvals from their insurance carriers. The program has become a comprehensive case management service made up of nurses and social workers who work with patients on many issues from access to coverage. Case managers also provide support to providers who are transitioning patients to home therapy. Home health agencies can benefit from the information and assistance offered by this program as well as other resources throughout Genzyme and the Gaucher community.
authors.forename: R S
authors.initials: RS
authors.lastname: Goldwater
mesh_terms: D019090:Case Management / Q000458:organization & administration*; D016527:Drug Costs; D004345:Drug Industry; D005776:Gaucher Disease / Q000188:drug therapy*; D005962:Glucosylceramidase / Q000191:economics / Q000627:therapeutic use*; D006801:Humans
chemical_list: C070292:alglucerase; D005962:Glucosylceramidase; C090568:imiglucerase