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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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Any content about investigational therapeutics or investigational uses of locally approved products does not establish the safety or efficacy of these therapeutics or uses, and there is no guarantee of local regulatory approval.
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Publications

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

Transthyretin Amyloidosis (ATTR)

Neurofilament light chain as a biomarker for hereditary ATTR amyloidosis

Amyloid

Author(s)

Masateru Tajiri, Mitsuto Sato, Minori Kodaira, et al

Acute Hepatic Porphyria (AHP)

Long-term follow-up of givosiran treatment in patients with acute intermittent porphyria from a phase 1/2, 48-month open-label extension study

Orphanet Journal of Rare Diseases

Author(s)

Eliane Sardh, Manisha Balwani, David C. Rees, et al

Acute Hepatic Porphyria (AHP)

High disease burden and healthcare resource usage in patients with acute porphyria—A population-based analysis

Liver International

Author(s)

Ulrich Stölzel, Bjoern Ambrosius, Brun S, et al

Primary Hyperoxaluria Type 1 (PH1)

Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 30-month analysis of the phase 3 ILLUMINATE-B trial

Frontiers in Pediatrics

Author(s)

Yaacov Frishberg, Wesley Hayes, Hadas Shasha-Lavsky, et al