Congenital Muscular Dystrophy (CMD)
Has the potential to treat multiple rare lysosomal storage disorders with neurological involvement, including GM1 and GM2 gangliosidoses, and Niemann-Pick disease type C (NPC).
Siklos is indicated for the prevention of recurrent painful vaso-occlusive crises including acute chest syndrome in adults, adolescents and children older than 2 years suffering from symptomatic sickle cell syndrome
Non-melanoma skin cancers and rare genetic skin manifestations.
