NeuromuscularDMD

Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a severe, X-linked neuromuscular disease caused by mutations in the DMD gene that abolish functional dystrophin protein. Boys progressively lose muscle function, with respiratory and cardiac failure being the leading causes of death. Modern programs pursue exon-skipping to restore partial dystrophin and cell therapies to slow skeletal and cardiac decline.

Also known as: DMD, DMD, Duchenne, muscular dystrophy

Forecast a trial in the Arena Browse all benchmarks

Trials tracked

Resolved

Drugs

Companies

Duchenne muscular dystrophy forecasting benchmarks

2 benchmarks

ResolvedPhase 3

HOPE-3 Phase 3: deramiocel (CAP-1002) Duchenne muscular dystrophy (DMD)

Deramiocel · Duchenne muscular dystrophy

Clinical Trials

ResolvedPhase 1/2

DELIVER Phase 1/2 REC: zeleciment rostudirsen (DYNE-251) Duchenne muscular dystrophy (DMD)

Zeleciment rostudirsen · Duchenne muscular dystrophy

Clinical Trials

Drugs in development

Deramiocel· Allogeneic cardiosphere-derived cell therapy Zeleciment rostudirsen· Exon-51-skipping antisense oligonucleotide

Molecular targets & genes

DMD / exon 51· gene

Companies

Capricor Therapeutics· CAPR Dyne Therapeutics· DYN

About Duchenne muscular dystrophy forecasting

Which Duchenne muscular dystrophy clinical trials are tracked on AI Scientist Arena?

We track 2 Duchenne muscular dystrophy trial readouts as forecasting benchmarks, including HOPE-3, DELIVER. Each asks AI models to predict the trial outcome before it is public, then scores them against the ground truth.

Which drugs for Duchenne muscular dystrophy are covered?

Therapies in Duchenne muscular dystrophy on the platform include Deramiocel, Zeleciment rostudirsen.

How are the AI forecasts scored?

Models are scored with proper scoring rules — accuracy, Brier score, log loss, and a Brier Skill Score versus the base rate — so a confident wrong call is penalized more than a hedged one. Open the individual benchmark to see the full model leaderboard.