[HoyaSaxa]

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.

[ribs]

My friend’s boy died just after entering college with it. Ugh.

[sdflhasjd]

It's definitely possible to survive past 25, that's just the average life expectancy.

[light_hue_1]

That doesn't sum it up at all.

Without ventilation almost everyone dies by 25 which was the case until the 70s. The poster is right there's a marked decline by your early 20s. And depending on when and where their story happened it could be very accurate: it was almost impossible to make it past 25.

Almost everyone needs to be on ventilation to make it further and then the median survival is just shy of 30. That significantly impacts your quality of life unfortunately.

[Traubenfuchs]

I think it would be better for the quality of discourse if people online would stop going into discussions about numbers like this without easily googleable sources.

Meta analysis from end of 2021:

https://n.neurology.org/content/97/23/e2304

"The median survival age from the pre-1970 birth cohort was 18.3 years (95% CI 18.0, 18.9) compared to 24.0 years in the 1970 to 1990 birth cohort (95% CI 22.8, 25.0) and 28.1 years in the post-1990 birth cohort (95% CI 25.1, 30.3)."

"The cohort analysis is also likely to represent a comparison of ventilated and nonventilated patients because ventilation was introduced in many clinical settings only in the 1990s."

[sdflhasjd]

Sorry, I'm unfortunately aware, a friend of mine succumbed to DMD at just 21 even with significant medical care. I was just taking issue with the rather absolute "impossible to survive".