PPMD Benefit/Risk Study Results Published in Clinical Therapeutics - PPMD Community

PPMD Benefit/Risk Study Results Published in Clinical Therapeutics - PPMD Community: "PPMD is thrilled that results from the recent benefit/risk project our team led have been published in Clinical Therapeutics. The article concludes that caregivers are willing to accept a serious risk when balanced with noncurative slowing or stopping of the progression of muscle weakness, even absent improvement in lifespan. These preferences should inform the Food and Drug Administration’s (FDA’s) benefit–risk assessment of emerging Duchenne therapies."



'via Blog this'