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US FDA lifts hold on Solid Biosciences' trial for muscle disease therapy

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US FDA lifts hold on Solid Biosciences' trial for muscle disease therapy

Solid Biosciences Inc. said the U.S. Food and Drug Administration removed the clinical hold on a trial evaluating its gene therapy SGT-001 as a treatment for Duchenne muscular dystrophy.

Duchenne muscular dystrophy, or DMD, is a genetic disorder characterized by progressive muscle degeneration and weakness.

The phase 1/2 trial, named Ignite DMD, was evaluating the safety and effectiveness of SGT-001, a gene therapy for treating children and adolescents with DMD.

Ignite DMD was put on hold by the FDA after a patient treated with the therapy was hospitalized due to a drop in platelet count, low blood cell count, transient renal impairment and signs of complement activation.

The company made changes to the trial protocol in connection with the lifting of the clinical hold.

Solid Biosciences said early data from a prespecified interim analysis of the trial will be reported in the second half of 2019.