Right Marijoh - there is one adequately-controlled double blind placebo trial (Reach2HD) for HD with PBT2 and two others. (SRPT has one open label trial). Many days of dosing with PBT2 and no SAEs, only one after someone went off the drug. PROs of improved cognition with PBT2 in HD.
Janet Woodcock was directing the panel that they should consider the comments by the advocates and patients and that they had flexibility when considering accelerated approval. Also, Dr. Temple was speaking of using historical controls as a comparison mechanism and that other trials had successfully employed their use to obtain approval like Colin Masters was trying to do.
Sarepta Rare Disease Advisory Panel Meeting today, page-7
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