Gaucher disease: a strategic collaborative approach from the European Medicines Agency and Food and Drug Administration
| Current version |
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| Reference number | EMA/237265/2017 |
| Published | 30/06/2017 |
| Keywords | Gaucher disease, lysosomal storage disorder, enzyme replacement therapy, substrate-reduction therapy |
| Description | This document aims to increase the chances of rapid and smooth agreement of the paediatric investigation plan (EMA)/pediatric study plan (FDA). It also discusses the possibility of a multi-arm, multi-company clinical trial for the treatment of Gaucher disease, as one approach to address the feasibility of developing multiple products for a rare disease in a limited timeframe. |
Document history
First version Current version |
| Published: 30/06/2017
In operation: 30/06/2017-present Published: 30/06/2017 Published: 14/05/2014 |