January 16, 1998

TO:	Families of Those Inquiring about Participation in Duchenne Muscular Dystrophy Gene Therapy Trials
RE:	Gene Therapy Research Projects
FROM:	Ronald J. Schenkenberger Director of Research and Patient Services Administration

Thank you for your recent inquiry about the ground-breaking gene therapy research being conducted by MDA-supported investigators at Ohio State University in collaboration with researchers at the University of Michigan. We welcome your submission of an application form*. In doing so, please keep in mind that the initial trials are designed to assess the safety of the procedure and to provide data that will allow larger scale trials to be undertaken. The initial trials do not represent a treatment for the disease but are designed to provide answers to questions that hopefully will lead to an expanded trial.

Researchers are targeting March of this year to begin screening youngsters ages 5, 6, and 7 and, pending FDA authorization, 12 will be selected in the initial trial to begin receiving injections by Fall of this year. While MDA is funding the project, the final decision regarding participation rests with the researchers conducting the study.

The budget for the project does not allow for reimbursement for travel and lodging related to participation in these initial trials. Participants will be expected to make at least monthly visits to Ohio State University in Columbus for at least the first year of the program.

Your completed application should be sent to researchers at Ohio State University, at the address shown on the application form. They will be responding to you directly.

RJS:mr

^*Web Site Editor's Note: To receive an application, please call the MDA national office at (800) 572-1717.