gene therapy trial

A phase 1 gene transfer trial in nine babies with type 1 spinal muscular atrophy has opened at Nationwide Children's Hospital in Columbus, Ohio

posted on May 6, 2014 - 9:33am
A phase 1 trial to test the safety and efficacy of gene transfer therapy in infants with type 1 spinal muscular atrophy (SMA) who are 9 months old or younger has opened at Nationwide Children's Hospital in Columbus, Ohio, under the direction of neurologist Jerry Mendell.

Investigators in Columbus, Ohio, need to determine the best way to assess thigh-muscle strength in men with BMD before conducting a gene therapy study

posted on January 18, 2011 - 4:32pm
A new study to determine the best "outcome measure" (measurable activity) with which to assess thigh-muscle (quadriceps) strength in men with Becker muscular dystrophy (BMD) is seeking participants. The study, taking place at Nationwide Children's Hospital in Columbus, Ohio, is a necessary prelude to a planned trial of gene therapy involving injections of genes for the follistatin protein in...
posted on May 1, 2006 - 3:05pm
QUEST Vol. 13, No. 3
MDA begins first U.S. Duchenne gene therapy trial