THE GREATEST GUIDE TO MUSCULAR DYSTROPHY THERAPIES VASANT KUNJ

not too long ago, the antibiotic gentamicin has long been tested in boys with DMD, about 10% of whom have inherited a dystrophin gene with a premature stop codon that halts the synthesis with the protein N-terminal into the dystroglycan binding location. Gentamicin suppresses chain termination, permitting some whole-length dystrophin to be produced