Rare Disease Clinical Trials in Türkiye

Amyotrophic lateral sclerosis

ALS in Türkiye: an estimated 4,000 living patients, 21 institutions with published experience in 6 cities, and no competing industry study yet. What sponsors should know before country selection.

Duchenne muscular dystrophy

Duchenne muscular dystrophy in Türkiye: over a hundred new diagnoses a year, 96 sites with trial or publication experience in 24 cities, 17 industry trials already run here. What sponsors should know before country selection.

Friedreich ataxia

Friedreich ataxia in Türkiye: an estimated 2,000 patients, an autosomal recessive disease enriched by consanguinity, 20 institutions with published experience and only one industry trial site so far. What sponsors should know.

Retinitis pigmentosa

Retinitis pigmentosa in Türkiye: an estimated 20,000 patients, a high share of autosomal recessive disease, 25 institutions with published experience and no industry trial site yet. What gene and cell therapy sponsors should know.

Idiopathic pulmonary fibrosis

Idiopathic pulmonary fibrosis in Türkiye: over 12,000 estimated patients, 144 sites with trial or publication experience in 25 cities, antifibrotics reimbursed. What sponsors should know before country selection.