Some good discussion points reader and that's why safety has been a key end point. Would you prefer a drug which is administered intravenously? I see that the drug being dosed orally as a massive positive in comparison. But that is only my opinion, I guess the family members and sufferers are ultimately the ones to ask.
The company has been very clear in stating they are working with the FDA, which may be for this exact reason. This, along with the limited treatment options, reduces the risk in my eyes.
Each to their own.
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