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Publications

Discover peer-reviewed publications by therapeutic area.
The information on this page pertains to select publication resources. It is not intended to serve as a comprehensive list of all publications resources.
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Publications

Transthyretin Amyloidosis (ATTR)

Treatment with vutrisiran in people with transthyretin amyloidosis with cardiomyopathy: A plain language summary

Future Cardiology

Author(s)

Marianna Fontana, Julian D Gillmore, Ronald M Witteles, et al

Primary Hyperoxaluria Type 1 (PH1)

Efficacy and Safety of Lumasiran for Advanced Primary Hyperoxaluria Type 1: 24-Month Follow-Up of the Phase 3 Illuminate-C Trial

American Journal of Kidney Diseases

Author(s)

Anne-Laure Sellier-Leclerc, Daniella Magen, Hadas Shasha-Lavsky, et al

Transthyretin Amyloidosis (ATTR)

Diagnosis of hereditary transthyretin amyloidosis in patients with suspected chronic inflammatory demyelinating polyneuropathy unresponsive to intravenous immunoglobulins: results of a retrospective study

Orphanet Journal of Rare Diseases

Author(s)

Yann Péréon, David Adams, Jean-Philippe Camdessanché, et al

Transthyretin Amyloidosis (ATTR)

Impact of Vutrisiran on Functional Capacity and Quality of Life in Transthyretin Amyloidosis with Cardiomyopathy

Journal of the American College of Cardiology

Author(s)

Farooq H. Sheikh, Gilbert Habib, W. H. Wilson Tang, et al

Transthyretin Amyloidosis (ATTR)

Impact of Heart Failure Severity on Vutrisiran Efficacy in Transthyretin Amyloidosis with Cardiomyopathy

Journal of the American College of Cardiology

Author(s)

Mathew S. Maurer, Ronald M. Witteles, Pablo Garcia-Pavia, et al

Acute Hepatic Porphyria (AHP)

Patient experience with acute hepatic porphyria before and after long-term givosiran treatment in a qualitative interview study

Molecular Genetics and Metabolism Reports

Author(s)

Hetanshi Naik, Michelle Brown, Stephen Meninger, et al

Transthyretin Amyloidosis (ATTR)

Worsening of Heart Failure in Outpatients With Transthyretin Amyloidosis and Cardiomyopathy in the APOLLO-B Trial

Journal of the American College of Cardiology

Author(s)

Marianna Fontana, Mathew S. Maurer, Julian D. Gillmore, et al

Transthyretin Amyloidosis (ATTR)

Five-Year Results With Patisiran for Hereditary Transthyretin Amyloidosis With Polyneuropathy

JAMA Neurology

Author(s)

David Adams, Jonas Wixner, Michael Polydefkis, et al

Transthyretin Amyloidosis (ATTR)

The journey to diagnosis of wild-type transthyretin-mediated (ATTRwt) amyloidosis: a path with multisystem involvement

Orphanet Journal of Rare Diseases

Author(s)

Chafic Karam, Colleen Moffit, Catherine Summers, et al

Transthyretin Amyloidosis (ATTR)

Outpatient Worsening Heart Failure in Patients with Transthyretin Amyloidosis with Cardiomyopathy in the HELIOS-B Trial

Journal of the American College of Cardiology

Author(s)

Marianna Fontana, Mathew S. Maurer, Julian D. Gillmore, et al