AlveoGene’s Novel, Inhaled Gene Therapy AVG-002 Receives Orphan Drug Designation from FDA for Lethal Neonatal Surfactant Protein B (SP-B) Deficiency
03 déc. 2024 04h00 HE
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Alveogene
Inherited SP-B deficiency is an ultra-rare monogenic cause of fatal respiratory distress syndrome in newborn infants with few, if any, options for long-term survivalHighly encouraging preclinical data...