Articles by tag: rare-diseases

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.

Vyjuvek gene gel treatment butterfly disease DEB 2026

UK approves Vyjuvek gene gel for wound treatment in dystrophic epidermolysis bullosa. Efficacy 67% at 6 months. Learn mechanism of action and prospects.