Patients

Understanding RNAi

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

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Learn About?How RNAi-Based Medicines Work

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Learn about RNAi and our investigational therapies

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

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Caregivers

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

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Interested in our clinical trials?

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Our Focus On You

A Family Living with
hATTR Amyloidosis
Get more information about hATTR Amyloidosis.
Ania: Living with
Porphyria
Get more information about porphyria.
A Family Living with
Primary Hyperoxaluria Type 1 (PH1)
Get more information about primary hyperoxaluria.

Access To Investigational Medicines

KU体育 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, KU体育 will consider providing a requesting physician with pre-approval access to a specific KU体育 investigational medicine, for the treatment of an individual patient outside of a clinical trial, when certain conditions are met.

KU体育 Assist?

KU体育 Assist? offers you a number of personalized services throughout your treatment with an KU体育 product.

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KU体育 Act?

Through KU体育 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 KU体育-sponsored clinical trials.

Science Of RNAi

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