Gene Therapy Trial Update
 
Dr. Mendell, the lead investigator on the follistatin gene therapy trial, is encouraged by the improvement seen in Becker muscular dystrophy patients who have been treated along with the sIBM patients in the follistatin gene therapy. He believes the results in the Becker patients have potential for sIBM patients. The Becker patients received a slightly higher dose of the treatment than the first sIBM patients. The results in the Becker patients might allow the next 6 sIBM patients to be treated with the highest dose of the treatment.  This should produce better results in the sIBM patients as well. Approval is now being sought from federal regulatory bodies to allow the higher dose treatment.  Should you have any questions about the trial or patient recruitment, please contact This e-mail address is being protected from spambots. You need JavaScript enabled to view it at Nationwide Children's. A firsthand account from one of the patients to be treated among the first group will be published in the upcoming issue of OutLook, TMA's quarterly newsletter.