Amyotrophic lateral sclerosis (ALS) is a progressively debilitating disorder of the central nervous system characterized by motor neuron degeneration. ALS causes progressive weakness and atrophy of…
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…