Our Priority Is to Put You at the Center

At Alnylam, we want to understand your journey, your experiences, and your unique needs. You inspire us to learn as much as we can, to persevere and continue to advance the development of potential therapies to treat rare and serious diseases. We take pride in working together to achieve mutual goals such as increasing awareness, enabling diagnosis, and providing education and services to support patient communities. We have a dedicated team to ensure your perspectives are represented at Alnylam. If you are an advocate interested in helping us increase awareness of rare diseases, please send an email to

Understanding RNAi

RNA interference (RNAi) is the core discovery that forms the therapies Alnylam is developing. It is recognized as a major scientific breakthrough—but how does it work, exactly?

Learn about How RNAi-based Medicines Work

Learn about RNAi and our investigational therapies

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Alnylam is focused on 4 Strategic Therapeutic Areas (STArs): genetic medicines, cardio-metabolic diseases, infectious diseases, and central nervous system (CNS) and ocular diseases

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Alnylam is dedicated to providing support for affected individuals, families, and caregivers

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Interested in our clinical trials?

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Learn More About Our Products


 Focus on You

A Family Living with
 hATTR Amyloidosis

Get more information about hATTR Amyloidosis.

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Ania: Living with

Get more information about porphyria.

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A Family Living with
Primary Hyperoxaluria Type 1 (PH1)

Get more information about primary hyperoxaluria.

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Access to
Investigational Medicines

Alnylam is dedicated to developing new therapies that have a positive impact on patient health, and to serving patients, patient families and patient communities through education, empathy, and awareness. We understand that there are seriously ill patients who will not be eligible for our clinical trials and may not have options for alternative therapies, including investigational therapies in trials being conducted by other sponsors. In these circumstances, Alnylam will consider providing a requesting physician with pre-approval access to a specific Alnylam investigational medicine, for the treatment of an individual patient outside of a clinical trial, when certain conditions are met.


Alnylam Assist® offers patients personalized support throughout their treatment with an Alnylam product.

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Through Alnylam Act®, healthcare professionals can request genetic testing and counseling for hATTR amyloidosis, acute hepatic porphyria, or primary hyperoxaluria type 1 for individuals meeting certain criteria.

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Learn more about Alnylam-sponsored clinical trials.

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RNAi is a breakthrough in the understanding of how genes are regulated in cells.

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