PPMD on benefit-risk assessments in rare disorders

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The case for therapeutic development in Duchenne Muscular Dystrophy as the prototype for new approaches

Pat Furlong of Parent Project Muscular Dystrophy kindly shared this whitepaper, ‘Benefit-Risk Assessments in Rare Disorders: The case for therapeutic development in Duchenne Muscular Dystrophy as the prototype for new approaches.’

“This paper examines the similarities and differences between benefit-risk assessments for common, progressive disorders vs. rare, progressive diseases.”

The paper goes on to explore:

Comprehensive conceptual and operational approaches to benefit-risk considerations
Considerations in rare disorder PFDD
Duchenne as a prototype for rare disorder benefit-risk assessment
Survey data regarding Duchenne views on benefit and risk
The case for Duchenne Muscular Dystrophy as the prototype for rare disease benefit-risk regulator decision making

To learn more about this download a copy today.

PPMD on benefit-risk assessments in rare disorders

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The case for therapeutic development in Duchenne Muscular Dystrophy as the prototype for new approaches