Aperture

Neuromuscular · Rare

Duchenne muscular dystrophy

Updated August 2026 · DMD (dystrophin, Xp21)

Algorithm

All patients — foundational

Multidisciplinary neuromuscular care, cardiac and respiratory surveillance, physiotherapy. Corticosteroid from ~2 years unless contraindicated.

Add-on, mutation-agnostic

Givinostat on top of steroids from age 6 where reimbursed. Does not replace SOC.

Mutation-specific chronic

Weekly PMO skippers if amenable and access exists. US-centric commercial reality.

One-time gene transfer

Only ambulatory ≥4 after the 2025 label reset. Centre-restricted. Informed consent now includes fatal ALF.

Foundational SOC

Prednisone / prednisolone

prednisone · Generic

List / typical annual

$400

Generic annual

Eligible
All DMD, typically from diagnosis / ~2y+
Route
Oral daily
Endpoint that matters
NSAA, 6MWT, time to LoA (historical / CINRG)
Curative?
Supportive
Efficacy
Delays loss of ambulation ~2–3 years vs natural history. Standard against which everything else is stacked.
Safety
Weight, bone density, stature, behaviour, cataracts, adrenal suppression. Lifelong management problem.
Residual unmet need
Does not restore dystrophin. Side-effect tax is the reason Agamree exists.