Long-Term Data Demonstrate Sustained Efficacy of Risdiplam in Spinal Muscular Atrophy
In Part 2 of the trial, patients 2-25 years of age received either risdiplam for 24 months or placebo for 12 months followed by risdiplam for 12 months.
In Part 2 of the trial, patients 2-25 years of age received either risdiplam for 24 months or placebo for 12 months followed by risdiplam for 12 months.
A New Drug Application (NDA) has been submitted to the Food and Drug Administration (FDA) for triheptanoin (Ultragenyx) for the treatment of long-chain fatty acid oxidation disorders.