Public Meeting on Inborn Errors of Metabolism Patient-Focused Drug Development

On June 10, 2014, FDA conducted a public meeting on Patient-Focused Drug Development for neurological manifestations of inborn errors of metabolism. FDA is interested in obtaining patient input on the impact of the neurological manifestations of inborn errors of metabolism on daily life and patient views on treatment options.


June 10, 2014


9:00 am to 1:00 pm


FDA White Oak Campus
10903 New Hampshire Ave.
Building 31, Room 1503A  (Great Room)
Silver Spring, MD 20993
(Information about arrival to FDA's White Oak campus)


To register for this meeting, visit: http://patientfocusediem.eventbrite.com
Registration closed on May 30, 2014.

Participants may be interested in a related workshop taking place on June 9, 2014 on the FDA White Oak Campus. For more information on this workshop, Immune Responses to Enzyme Replacement Therapies, go to: http://www.fda.gov/Drugs/NewsEvents/ucm392641.htm.

Submitting comments to the docket: In addition to providing input at the public meeting, patient stakeholders are invited to provide their perspectives on the discussion questions through the public docket. The docket closes on August 11, 2014.



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