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Perhaps, but the cost will be prohibitive for any off label use.
Not necessarily, it depends on what the patient is being treated for. Any of the rare diseases come into play. Not to mention that DMD affects upwards of 6,000 people, provides ample revenue without even considering off label use.
homozygous familial hypercholesterolemia - not DMD... My keyboard shortcut for hfh got auto-corrected instead of substituted.