Steven Winesett

A Phase 3 Randomized, Placebo-controlled, Double-blind Study to Evaluate the Efficacy and Safety of BBP-418 (ribitol) in Patients with Limb Girdle Muscular Dystrophy 2I (LGMD2I)

CTOA

Compassionate Use Program for olipudase alfa enzyme replacement therapy for patients with chronic acid sphingomyelinase deficiency (ASMD)

Phase 1b, Multi-center, Randomized, Double-Blind, Placebo-Controlled, Rising Multiple Dose Study to Evaluate the Safety, Tolerability, Pharmacodynamics and Pharmacokinetics of VK0214, in Subjects with the Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD)

Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) in Patients with Fabry Disease

Effects of Cannabidol Use on the Developing Brain in Medically Refractory Childhood Epilepsy