We have just published a MARA Rating for a one-time gene therapy for a rare childhood disease.

The trials answered one question clearly: do treated patients do better than untreated historical cases? They do. The outcomes were compelling.

Payers ask a different question: how does this treatment compare to existing alternatives — in outcomes, and in cost per patient?

That question was not addressed. No head-to-head trial exists. No quality-of-life data was collected. The cost exceeded standard thresholds.

Two questions. One answered. One not.

Read the full MARA Rating here: https://mararating.com/report/zolgensma-onasemnogene-abeparvovec-for-in-5q-spinal-muscular-atrophy-as-of-may-2026/