Committee for Orphan Medicinal Products: Public summary of positive opinion for orphan designation of retroviral gamma c cDNA containing vector for the treatment of severe combined immunodeficiency (SCID)-Xl disease.

On 30 May 2001, orphan designation (EU/3/01/038) was granted by the European Commission to Genopoietic SAS, France, for retroviral gamma c cDNA containing vector for the treatment of severe combined immunodeficiency (SCID)-Xl disease. On 15 May 2009, the European Medicines Agency's (EMEA) Committee for Orphan Medicinal Products (COMP) published a public summary of positive opinion for orphan designation of retroviral gamma c cDNA containing vector for the treatment of severe combined immunodeficiency (SCID)-Xl disease. There are questions and answers covering: What is severe combined immunodeficiency (SCID)-Xl disease?, What is the estimated number of patients affected by the …