Rahim R. Shukurov

Analytical Characterization, Pharmacokinetics, Safety, and Preliminary Immunogenicity of GNR-062, an Agalsidase Beta Biosimilar Candidate, in Healthy Male Volunteers

Fabry disease is a rare genetic multisystemic lysosomal disease characterized by progressive cellular accumulation of globotriaosylceramide (GL-3) in various organs with specific cutaneous (angiokeratoma), neurological (pain), renal (proteinuria, chronic kidney failure), cardiova...

Comparative Analysis of SMN2 Splicing Activity and Protein Production Following In Vitro Treatment with the Generic Risdiplam Drug Vapromin® and the Reference Drug Evrysdi®

Background/Objectives: Risdiplam is a low-molecular-weight small-molecule modifier of SMN2 pre-mRNA splicing that was developed for spinal muscular atrophy (SMA) therapy and approved for the treatment of SMA as Evrysdi® (Roche, Basel, Switzerland). Vapromin® is a generic drug pro...