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?

From Possibility to Patients: Understanding RNAi

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, hepatic 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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Alnylam supports eligible individuals with the Early Access Program aimed to help those living with hATTR amyloidosis, acute hepatic porphyria (AHP) or primary hyperoxaluria type 1 (PH1) gain access to investigational therapies. Speak with your medical professional to learn more about the programs and your eligibility.



A Family Living with
 hATTR Amyloidosis

Get more information about hATTR Amyloidosis.

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Candace: 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.


Interested in learning about our hATTR amyloidosis studies? Get started here.

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Alnylam Assist® helps patients access ONPATTRO® (patisiran) with personalized support.
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Alnylam Act provides no-charge, independent genetic testing and counseling to individuals in the US and Canada who may have hATTR amyloidosis, acute hepatic porphyria, or primary hyperoxaluria type 1 (PH1).

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Learn more about an FDA-approved treatment option available for patients in the U.S.
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