Patients
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Learn about RNAi and our investigational therapies

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Alnylam is focused on several disease areas including transthyretin amyloidosis, rare diseases, cardiovascular diseases, metabolic diseases, and neurological diseases.

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Caregivers

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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About RNAi Therapeutics and How They Work

RNAi therapeutics are a type of gene-silencing medicine invented by Alnylam scientists that is based on RNA interference (RNAi). The discovery of RNAi was awarded the Nobel Prize in Physiology or Medicine in 2006.

What is an RNAi therapeutic?

How do RNAi therapeutics treat disease?

How can unwanted proteins cause or contribute to disease?

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®

Alnylam Assist® offers you a number of personalized services throughout your treatment with an Alnylam product.

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Alnylam Act®

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.

Clinical Trials

Learn more about Alnylam-sponsored clinical trials.

Science Of RNAi

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