// App-Quantinova.ai

EP694 : IMPACT OF OLIPUDASE ALFA ENZYME REPLACEMENT THERAPY ON PULMONARY, VISCERAL AND HEMATOLOGIC MANIFESTATIONS OF ACID SPHINGOMYELINASE DEFICIENCY: 1-YEAR RESULTS OF CLINICAL TRIALS IN CHILDREN AND ADULTS

Researchers

Presenter

  • Maurizio Scarpa

Principal Investigators

  • Antonio Barbato

  • George A. Diaz

  • Renata C. Gallagher

  • Roberto Giugliani

  • Norberto B. Guelbert

  • Nathalie Guffon

  • Carla Hollak

  • Takayuki Ikezoe

  • Simon A. Jones

  • Robin Lachmann

  • Olivier Lidove

  • Paulina Mabe

  • Eugen Mengel

  • Ebubekir Senates

  • Michel Tchan

  • Jesus Villarrubia

  • Melissa Wasserstein

  • Yixin Chen

  • Sandy Furey

  • Catherine Ortemann-Renon

  • Qi Zhang

  • Monica Kumar

Medical Centers

  • Sanofi Genzyme, Cambridge, MA, United States

  • Department of Neonatology, Westmead Hospital, Sydney, NSW, Australia

  • Department of Chest Surgery, Fukushima Medical University, Fukushima, Japan

  • Charles Dent Metabolic Unit, National Hospital for Neurology & Neurosurgery London United Kingdom

  • Department of Clinical Medicine and Surgery, Federico II University, Naples, Italy

  • Clinica Santa Maria, Santiago, Chile

  • Sección Enfermedades Metabólicas,Hospital de Niños de la Santísima Trinidad,Córdoba,Argentina

  • St. Mary’s Hospital,Manchester University National Health Service Trust,Manchester,United Kingdom

  • Sanofi Genzyme,Bridgewater, NJ,United States

  • Mount Sinai Icahn School of Medicine, New York, New York, United States

  • Istanbul Medeniyet University Hospital, Istanbul, Turkey

  • UFRGS and INAGEMP,Porto Alegre,Brazil

  • Institute of Clinical Science for Lysosomal Storage Disorders,SphinCS GmbH,Hochheim,Germany

  • Regional Coordinator Centre for Rare Diseases,University Hospital of Udine,Udine,Italie

  • SphinCS,Hochheim,Germany

  • Endocrinology and Metabolism,Amsterdam UMC, University of Amsterdam,Amsterdam,Nederländerna

  • Centre de Référence des Maladies Rénales Rares Hôpital Femme Mère Enfant, Hospices Civils de Lyon Lyon France

  • Rheumatology Department, Hospital Universitario Ramόn y Cajal, Madrid

  • Dept of Neurology, University of California San Francisco, San Francisco, CA, United States

  • Childrens Hospital at Montefiore, Albert Einstein College of Medicine, Bronx, NY

  • Croix Saint-Simon Hospital, Paris, France

Locations

  • United States

  • Australia

  • Japan

  • United Kingdom

  • Italy

  • Chile

  • Argentina

  • Turkey

  • Brazil

  • Germany

  • Netherlands

  • France

  • Spain

Companies

  • N/A

Study Components

Therapeutic Area

  • Genetic Disorder

  • Endocrine/metabolic Diseases (ME)

  • Ophthalmology (OP)

Disease

  • Niemann-Pick disease type A

  • Anterior segment dysgenesis 1

Biomarkers

  • N/A

Drug/Treatment

  • N/A

Outcome

  • N/A

Study Design

  • Randomized

  • Placebo Control

  • Double Blind/Blinded

  • Open Label

  • Multicenteric

  • Controlled

Phase

  • II/III

Study Id's

  • NCT02004691/

  • NCT02292654/

Sponsors

  • N/A

Result

  • N/A

Copyright © 2026. QuantiNova All rights reserved