Refocusing an Existing PRO Instrument for Better Target Treatment Benefit in Neuromuscular Rare Disease

Urgent unmet need to develop and evaluate new therapies for degenerative pediatric neuromuscular rare disease

• Customer previously developed a patient reported outcome (PRO) instrument for Spinal Muscular Atrophy (SMA), but it was too general to detect signals of treatment benefit in Non-Ambulatory SMA clinical trials.
• Can we refocus this PRO instrument using the customer’s existing in-house data?

Use a combination of traditional and state-of-the-art psychometric analytics to identify the best PRO items to form a new instrument.