March 2026
March 2026 may be remembered as one of the most consequential months rare disease medicines has seen in recent years. Four FDA approvals landed in the span of three weeks — each representing a first of its kind: the first gene therapy for LAD-I, the first enzyme replacement to cross the blood–brain barrier, the first […]
February 2026
Every year, Rare Disease Day (last day of February) highlights the significant challenges faced by patients living with rare conditions. Although each rare disease affects a small number of individuals, collectively they impact hundreds of millions worldwide. Many of these patients are children, and the effects of these diseases extend far beyond the patients themselves, […]
January 2026
Starting 2026 with a retrospective look at FDA approvals shows orphan drugs holding their ground as the backbone of biopharma innovation — accounting for roughly half of all new approvals over the past three years (28 of 55 in 2023, 26 of 50 in 2024, and 23 of 46 in 2025). 60% of 2025’s orphan […]
Ampleia Health Innovation Summit 2026
Une journée dédiée à connecter science, innovation et investissement pour accélérer les solutions en santé.
Réinventer l’innovation en santé : pourquoi les maladies rares changent la donne
Les maladies rares redéfinissent aujourd’hui les modèles d’innovation en santé, en conciliant impact clinique et création de valeur.