The Natural History of Mucolipidosis Type IV
Fabry Screening Study
The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease
018-706 A One Visit Follow Up of Adults with Fabry Disease who Started Long-term Enzyme Replacement Therapy As Children
LEAP Adult Gaucher A 156-week, two-part, open-label, multicenter, multinational study of the safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of GZ/SAR402671 in combination with Cerezyme in adult patients with Gaucher disease type 3
MODIFY. A multicenter, double-blind, randomized, placebo controlled, parallel-group study to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease.
PB-102-F50. A Phase 3, Open Label, Switch Over Study to Assess the Safety, Efficacy and Pharmacokinetics of pengunigalsidase alfa (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks for 52 weeks in Patients with Fabry Disease Currently Treated with Enzyme Replacement Therapy: Fabrazyme® (agalsidase beta) or Replagal (agalsidase alfa)
F20 Balance Study A Randomized, Double blind, Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated with Agalsidase Beta – Study Number PB-102-F20
Dr. Schiffmann has contributed over 260 peer-reviewed articles in national and international publications that have paved the way for increased knowledge of metabolic diseases and the methods used to diagnose and treat them.
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