Availability of data and materials
RARE DISEASE
Qualified researchers may request access to related clinical study documents. Please send your data sharing requests to datasharing@agios.com. The following considerations will be taken into account as part of the review: 1. Ability for external researcher to re-identify trial participants such as small rare disease trials or single-center trials. 2. Language used in data and requested documents (eg, English or other). 3. Informed consent language with respect to allowance for data sharing. 4. Plan to re-evaluate safety or efficacy data summarized in the approved product labeling. 5. Potential conflict of interest or competitive risk.