Quick Facts Field Details Active Ingredient Ivacaftor Drug Category Rare / Orphan Diseases Primary Use Cystic fibrosis (CFTR potentiator, specific mutations) Availability Available in Turkey ✓ Introduction Ivacaftor is a...
Quick Facts Field Details Active Ingredient Lumacaftor/Ivacaftor Drug Category Rare / Orphan Diseases Primary Use Cystic fibrosis homozygous F508del (CFTR modulator) Availability Available in Turkey ✓ Introduction Lumacaftor/Ivacaftor is a...
Quick Facts Field Details Active Ingredient Vestronidase alfa Drug Category Rare / Orphan Diseases Primary Use MPS type VII (Sly syndrome) — enzyme replacement Availability Available in Turkey ✓ Introduction...
Quick Facts Field Details Active Ingredient Eliglustat Drug Category Rare / Orphan Diseases Primary Use Gaucher disease type 1 (oral substrate reduction) Availability Available in Turkey ✓ Introduction Eliglustat is...
Quick Facts Field Details Active Ingredient Sebelipase alfa Drug Category Rare / Orphan Diseases Primary Use Lysosomal acid lipase deficiency (Wolman disease) Availability Available in Turkey ✓ Introduction Sebelipase alfa...
Quick Facts Field Details Active Ingredient Cerliponase alfa Drug Category Rare / Orphan Diseases Primary Use CLN2 Batten disease (intrathecal enzyme replacement) Availability Available in Turkey ✓ Introduction Cerliponase alfa...
Quick Facts Field Details Active Ingredient Alglucosidase alfa Drug Category Rare / Orphan Diseases Primary Use Pompe disease (glycogen storage disease type II) — ERT Availability Available in Turkey ✓...
Quick Facts Field Details Active Ingredient Avalglucosidase alfa Drug Category Rare / Orphan Diseases Primary Use Pompe disease (next-gen ERT) Availability Available in Turkey ✓ Introduction Avalglucosidase alfa is a...
Quick Facts Field Details Active Ingredient Elosulfase alfa Drug Category Rare / Orphan Diseases Primary Use MPS type IVA (Morquio A syndrome) — enzyme replacement Availability Available in Turkey ✓...
Quick Facts Field Details Active Ingredient Laronidase Drug Category Rare / Orphan Diseases Primary Use MPS type I (Hurler syndrome) — enzyme replacement Availability Available in Turkey ✓ Introduction Laronidase...