Several clinical studies related to the natural history of different LSD and early and late phase clinical trials in collaboration with different pharmaceuticals. Most subjects are evaluated and recruited for the studies in the clinic.
This is an extension of previous phase I/II clinical trial to continue to assess the safety, tolerability and exploratory efﬁcacy parameters of PRX-102 in adult patients with Fabry disease
This is an Expanded Access Program for olipudase alfa enzyme replacement therapy for patients with chronic acid sphingomyelinase deficiency (ASMD). Its a single arm, open label, repeated dose design for patients with ASMD, a severe, life threatening disease, during the period between completion of the clinical trials and receipt of a final response from the applicable regulatory authority.