Glaucoma is a progressive disease of the optic nerve caused by high intraocular pressure (IOP); it is one of the leading causes of vision loss in the United States and Europe. The majority of…
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…
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…