Sickle cell disease (SCD) is a rare genetic blood disorder characterized by polymerization of hemoglobin in red blood cells that distorts them into a sickle shape. This sickling leads to various…
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…
Sjögren’s syndrome (SS) is a progressive autoimmune disease characterized by chronic inflammation and lymphocytic infiltration of the exocrine glands; the disease may occur alone (primary SS) or…