Na stronie:
www.mda.org/
znalazłam taki artykuł:
PTC124 to Be Tested at Higher Doses in DMD
PTC Therapeutics of South Plainfield, N.J., will test a higher dose of its
experimental compound PTC124 in approximately 12 boys with Duchenne muscular
dystrophy (DMD) caused by a premature stop codon mutation. The drug is
designed to coax muscle cells to ignore these mutations and make functional
dystrophin.
In October, the company announced that, in a trial of 26 boys with D...