I received several emails related to “off-label use.” “Off-label use” means that an FDA-approved drug is used for an unapproved indication or in an unapproved age group, unapproved dosage, or unapproved form of administration. Once a drug has been approved for one indication, physicians are free to prescribe it for any other indications that in their professional judgment is both safe and effective.
We are working very hard to identify the best FDA-approved drug that can potentially delay the progression of Wolfram. The reason I have not provided the details of our candidate drugs is that you could ask your physician to prescribe any of those candidate drugs for off-label use. I understand that the time of waiting is the hardest part of anything. I read the blog written by one of our patients, Adam Zwan. We need to bring you a safe treatment, but I also understand that the timing is critical.