36 BOYS NEEDED FOR EXPANDED STUDY OF GENTAMICIN IN DUCHENNE MD

An MDA-supported study of the antibiotic gentamicin (Garamycin) to treat Duchenne muscular dystrophy (DMD) is recruiting 36 boys with that disorder. The selected boys will be given intravenous gentamicin every three days for six months.

Participants must have a type of genetic mutation called a premature stop codon in the gene for the protein dystrophin, rather than a deletion, the more common type of gene flaw in Duchenne dystrophy. Study candidates will be directed to a center where they can obtain specialized testing for this mutation.

Two recent studies of gentamicin in people with Duchenne, Becker and limb-girdle muscular dystrophies yielded confusing results (Quest, vol. 8, no. 3). Studies in mice have suggested that gentamicin might coax cells into producing full-length proteins whose production would otherwise be interrupted by stop codons.

This larger study seeks to clarify earlier results and to determine whether gentamicin should be pursued as a treatment for Duchenne MD or other genetic disorders.

For further information, contact Cheryl Wall, trials coordinator at Ohio State University in Columbus, at (614) 293-9016 or at wall.49@osu.edu.