Articles by tag: orphan-drugs

EMA approved the first therapy for PROS: analysis of Vijoice

Despite the failure of the confirmatory study, EMA approved alpelisib for severe PROS syndrome. We analyze the mechanism, risks, and prospects of Vijoice.

Gene therapy AB-1009 for Pompe disease: clinical trial

A clinical trial of gene therapy AB-1009 for late-onset Pompe disease (LOPD) has begun. Phase 1 analysis, mechanism of action, risks, and prognosis. Learn more.

Marne-Cel FDA approval: gene therapy for LAD-I

FDA approved Marne-Cel for children with severe LAD-I: 100% survival in trials. Learn how Rocket Pharmaceuticals turned rejections into a breakthrough.

Innovative therapy for hemophilia and Fabry disease in the High-Cost Nosologies program

The Ministry of Health Commission will consider lonocotocog alfa and pegunigalsidase alfa for the High-Cost Nosologies program. Find out why inclusion is important for patients and what obstacles have arisen.