Geographic atrophy (GA), an advanced form of dry age-related macular degeneration (AMD), is a leading cause of visual impairment and blindness in the United States and Europe. Degradation of cells…
Idiopathic pulmonary fibrosis (IPF) is a chronic lung disorder characterized by an irreversible loss of lung function. Roche’s pirfenidone (Esbriet) was the first disease-modifying therapy (DMT)…
Muscular dystrophy (MD) is a spectrum of genetic disorders characterized by muscle weakness that, in severe forms, can lead to loss of ambulation and early mortality. Treatments are needed that can…
Approximately 20% of Western patients with nonsquamous non-small-cell lung cancer (NSCLC) present with EGFR mutations, creating a market for targeted and personalized treatments. Although the…
Myelodysplastic syndromes (MDS) are a heterogeneous collection of hematopoietic stem cell disorders that result in cytopenias and the risk of progression to acute myeloid leukemia (AML). Although…