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…
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…