posted on October 1, 2010 - 12:25pm
Featured in this issue: First U.S. exon-skipping trial opens * Utah researchers seek families with Ullrich CMD or Bethlem myopathy * Acceleron receives Fast Track, Orphan Drug designations for ACE031 * BioMarin abandons BMN195 development


posted on March 31, 2010 - 2:56pm
QUEST Vol. 17, No. 2
Featured in this issue: Disappointing results from ataluren trial leads to further analysis * First U.S. site for idebenone in DMD trial opens * Utrophin-boosting drug begins safety tests * Participants sought for trials in DMD, CMD, myotonic distrophy and periodic paralysis Study of PGD seeks couples who said 'no'