Medical Professionals
INFORMATION FOR MEDICAL PROFESSIONALS
KU体育 is committed to supporting and partnering with the healthcare community across the disease areas in which we work. We actively engage with medical professionals through our Medical Affairs team to provide information about our research, treatments in development, access to investigational medicines and more.
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What are RNAi Therapeutics and How do They Work?
RNAi therapeutics are an innovative and clinically-validated new class of medicines that use RNA interference (RNAi) to "silence" or turn off the production of specific genes that cause or contribute to disease. Our medicines utilize specially designed siRNA (small interfering RNA) therefore "interfering" with gene transcription. The world's first RNAi therapeutic (and our first commercial medicine) was approved in 2018 by U.S. and European regulators and several additional KU体育-developed medicines have been approved since.
LEARN MORE ABOUT RNAi THERAPEUTICS ?
Looking for information on our approved therapies? Visit the Our?Products page.
Our Pipeline
Our robust pipeline of investigational RNAi therapeutics is currently focused on several disease areas including transthyretin amyloidosis, rare diseases, cardiovascular diseases, metabolic diseases, and neurological diseases.
Late Stage
Learn more about our investigational therapies in hereditary ATTR amyloidosis, hemophilia, complement mediated diseases, and severe primary hyperoxaluria type 1.
Early Stage
Learn more about our investigational therapies across multiple disease areas.
KU体育 Grant Information
KU体育 is committed to supporting the medical and scientific understanding of our therapeutic areas of interest for healthcare professionals. Additionally, we are committed to supporting educational programs and initiatives spearheaded by organizations focused on patient advocacy, community education and support, and other scientific efforts related to our therapeutic areas of interest. To learn more about grants, sponsorships, or how to submit a grant request, visit our?grants page.
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.
To read our statement on access to investigational medicines,?click here.
Clinical Trials
Learn more about KU体育-sponsored clinical trials your patients may be eligible for.
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.
The Science Of RNAi
RNAi has helped create a new approach to developing medicine for genetic diseases.