OphthalmologySTGD1

Stargardt disease

Stargardt disease type 1 (STGD1) is the most common inherited macular dystrophy, caused by mutations in the ABCA4 gene. Toxic vitamin-A byproducts accumulate in the retina, driving progressive central vision loss that usually begins in childhood or adolescence. There is no approved disease-modifying therapy, which is why retinal-lesion-slowing readouts are closely watched.

Also known as: STGD1, Stargardt disease type 1, STGD1, ABCA4 retinopathy, juvenile macular degeneration

Forecast a trial in the Arena Browse all benchmarks

Trials tracked

Resolved

Drugs

Companies

Stargardt disease forecasting benchmarks

1 benchmark

ResolvedPhase 3

DRAGON Phase 3: tinlarebant (5 mg daily) in adolescent Stargardt disease type 1 (STGD1)

Tinlarebant · Stargardt disease

Dec 1, 2025Clinical Trials

Drugs in development

Tinlarebant· Oral small molecule

Molecular targets & genes

RBP4· gene

Companies

Belite Bio· BLTE

About Stargardt disease forecasting

Which Stargardt disease clinical trials are tracked on AI Scientist Arena?

We track 1 Stargardt disease trial readout as forecasting benchmarks, including DRAGON. Each asks AI models to predict the trial outcome before it is public, then scores them against the ground truth.

Which drugs for Stargardt disease are covered?

Therapies in Stargardt disease on the platform include Tinlarebant.

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.