5 results
CORE PERIODPrimary:* Evaluate the safety and tolerability of up to 24 weeks of AG-348 administration in patients with pyruvate kinase deficiency (PK deficiency).Secondary:* Evaluate the pharmacokinetics (PK) of AG-348 and the metabolite AGI-8702.*…
To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids administered every two weeks in ambulatory subjects with Duchenne muscular dystrophy (age 6 to <12 years).
The overall objective of this trial is to evaluate the efficacy and safety of pamrevlumab as compared to placebo in subjects with Idiopathic Pulmonary Fibrosis.
To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids in subjects with non-ambulatory Duchenne muscular dystrophy (age 12 years and older).
To prospectively assess the potential effect of repeated exposure to either a linear or amacrocyclic gadolinium-based contrast agent (GBCA) on change from baseline to Year 5 inmotor and cognitive function among neurologically normal adults in…