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: HELIOS A Results from the Phase 3 Study of Vutrisiran in Patients with Hereditary Transthyretin Mediated Amyloidosis with Polyneuropathy

Researchers

Presenter

  • David Adams

Principal Investigators

  • Ivailo Tournev

  • Mark Taylor

  • Teresa Coelho

  • Violaine Plante-Bordeneuve

  • John Berk

  • Alejandra Gonzalez-Duarte

  • Julian Gillmore

  • Soon-Chai Low

  • Yoshiki Sekijima

  • Laura Obici

  • Chongshu Chen

  • Prajakta Badri

  • Seth Arum

  • John Vest

  • Michael Polydefkis

Medical Centers

  • Neurology Department, APHP, CHU Bicêtre, INSERM U1195, Université Paris-Saclay

  • University Hospital Aleksandrovska & New Bulgarian University

  • Westmead Hospital

  • Centro Hospitalar Universitário do Porto

  • CHU Henri Mondor – Assistance Publique Hopitaux de Paris

  • Boston Medical Center, Amyloidosis Center, Boston University

  • Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán,...

  • University CollegeLondon, UK

  • University Malaya Medical Centre

  • Department of Medicine (Neurology and Rheumatology), Shinshu University School of Medicine, Matsumoto, Japan

  • Oncology, Fondazione IRCCS Policlinico San Matteo,

  • Alnylam Pharmaceuticals

  • The Johns Hopkins University School of Medicine, MD.

Locations

  • United States

  • Portugal

  • United Kingdom

Companies

  • N/A

Study Components

Therapeutic Area

  • Genetic Disorder

  • Peripheral Nervous System

Disease

  • Peripheral neuropathy

  • Amyloid disease

  • Familial Amyloid Polyneuropathy

Biomarkers

  • Transthyretin

Drug/Treatment

  • Patisiran

Outcome

  • N/A


Study Design

  • Randomized

  • Placebo Control

Phase

  • III

Study Id's

  • NCT03759379

Sponsors

  • N/A

Result

  • N/A