Quick Facts Field Details Active Ingredient Idursulfase Drug Category Rare / Orphan Diseases Primary Use MPS type II (Hunter syndrome) — enzyme replacement Availability Available in Turkey ✓ Introduction Idursulfase...
Quick Facts Field Details Active Ingredient Agalsidase beta Drug Category Rare / Orphan Diseases Primary Use Fabry disease (enzyme replacement therapy) Availability Available in Turkey ✓ Introduction Agalsidase beta is...
Quick Facts Field Details Active Ingredient Migalastat Drug Category Rare / Orphan Diseases Primary Use Fabry disease — amenable mutations (pharmacological chaperone) Availability Available in Turkey ✓ Introduction Migalastat is...
Quick Facts Field Details Active Ingredient Velaglucerase alfa Drug Category Rare / Orphan Diseases Primary Use Gaucher disease (enzyme replacement therapy) Availability Available in Turkey ✓ Introduction Velaglucerase alfa is...
Quick Facts Field Details Active Ingredient Agalsidase alfa Drug Category Rare / Orphan Diseases Primary Use Fabry disease (enzyme replacement therapy) Availability Available in Turkey ✓ Introduction Agalsidase alfa is...
Quick Facts Field Details Active Ingredient Imiglucerase Drug Category Rare / Orphan Diseases Primary Use Gaucher disease (enzyme replacement therapy) Availability Available in Turkey ✓ Introduction Imiglucerase is a Rare...
Quick Facts Field Details Active Ingredient Onasemnogene abeparvovec Drug Category Rare / Orphan Diseases Primary Use SMA type 1 (gene therapy, one-time IV) Availability Available in Turkey ✓ Introduction Onasemnogene...
Quick Facts Field Details Active Ingredient Miglustat Drug Category Rare / Orphan Diseases Primary Use Gaucher disease type 1, Niemann-Pick type C (substrate reduction) Availability Available in Turkey ✓ Introduction...
Quick Facts Field Details Active Ingredient Nusinersen Drug Category Rare / Orphan Diseases Primary Use Spinal muscular atrophy (SMA) — antisense oligonucleotide Availability Available in Turkey ✓ Introduction Nusinersen is...
Quick Facts Field Details Active Ingredient Risdiplam Drug Category Rare / Orphan Diseases Primary Use Spinal muscular atrophy (SMA) — oral SMN2 splicing modifier Availability Available in Turkey ✓ Introduction...