How should we measure quality of life impact in rare disease? Recent learnings in spinal muscular atrophy

How should we measure quality of life impact in rare disease? Recent learnings in spinal muscular atrophy The measurement of quality of life in the context of spinal muscular atrophy (SMA) is challenging. This is because the disease is experienced by children and is rare, which makes data collection difficult. This briefing reports on a symposium that outlined some lessons that can be learnt from the SMA context that might be more widely applicable to other rare diseases. Office of Health Economics