I appreciate that most people in this thread are not intimately familiar with this disease, but the casual attitude to it is woefully missing it’s savageness.

It unfortunately took my nephew’s good friends life at 13 years of age. You would have never known he had a disease until well past the age of 5. His family provided the most cutting age treatments available including those not yet FDA approved. Yet he deteriorated rapidly between the ages of 10 and 13.

For those that don’t know, it is almost impossible to survive past 25 with DMD

I cannot speak to the efficacy of this particular drug, but I will optimistically celebrate its approval in hopes that no other family has to watch their child suffer.