Aperture

Neuromuscular · Rare · teaser

Spinal muscular atrophy

Updated August 2026 · SMN1 (5q)

Teaser case. Selected price and peak-sales fields are held for a paid Brief — the rest is the method.

← Pipeline

Confidential in teaser · Biologic · Phase 2

Muscle-directed add-on

Thesis

Stack on SMN restoration to buy residual function. The only remaining $1B+ SMA question.

Probability of success

held

  • Additive function on top of SMN Rx▲ High
  • Trial in a treated population (harder bar)▼ High

Time to market

held

  • —Available on Brief

Peak revenue (US+EU+JP dir.)

held

Peak build

  • Peak build held

What kills the number

  • If the functional delta is small, payers will not stack a fourth SMA drug.