FOM 5.5.3

Question 1

Treatment of DMD doesn’t cure it, but does what?

Answer 1

Exon skipping to have partially active protein. This will make it duchenne to becker which is less serious

Question 2

What is characteristic of beckers muscular dystrophy?

Answer 2

Reduced number of spectrin repeats

Question 3

What is characteristic of duchennes?

Answer 3

Loss of c-terminal dystroglycan