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Patients & Community

Ensuring patients get the information they need

Since the beginning, we have been focused on patient needs across a wide variety of diseases. The guidance, collaboration and expertise of the patient communities we serve play a critical role in our success.

We believe in maintaining open and direct communications with the advocacy community. We work with partners, collaborators, organizations, and societies with similar goals to make sure patients are getting the support and resources they need. The following are some of our partners.

Ionis Clinical Trials

Before any treatment is approved for human use by the FDA and other regulatory authorities around the world, it must first prove to be safe and effective in clinical trials.

Ionis clinical trials

Our trials are designed with the greatest needs of the patients who will benefit from them in mind. They reflect input from patient communities as well as healthcare providers and regulatory agencies who share our commitment to providing access to safe and effective therapies as soon as possible.

We have many ongoing clinical trials designed to evaluate our medicines in a variety of areas of unmet medical need, including cardiovascular, metabolic, severe and rare diseases, including neurological disorders and cancer.

Learn more about Ionis clinical trials


For answers to questions you may have, connect with us.

Expanded access

Ionis Pharmaceuticals is driven by the fact that sick people depend on us, so we work tirelessly to ensure that our first-in-class and/or best-in-class medicines get to the patients in the most efficient and quickest way possible. One of the first steps we take is to conduct clinical trials. During clinical trials, we collect safety and efficacy data about our medicines in development with the goal of submitting those data to regulatory authorities, like the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), so that these medicines can be approved for use by patients.

Participation in one of our clinical trials is generally the most appropriate way to access our medicines in development. However, in very rare circumstances, Ionis Pharmaceuticals can make these medicines available for patients outside of a traditional clinical trial under an expanded access protocol, sometimes referred to as a treatment protocol. This is a program where patients may receive the medication if they meet certain criteria and are informed about the risks of participation. Ionis Pharmaceuticals is not currently considering individual requests for expanded access that are outside of a company-run expanded access protocol.

In deciding whether to establish an expanded access protocol, Ionis will evaluate whether:

  • There is substantial scientific evidence to support both the safety and the efficacy of the medication for the proposed use, typically after positive Phase 3 data is available;
  • It has been established that expanded access will not interfere with the conduct of clinical trials or other aspects of development that could impact marketing approval;
  • There is adequate supply of the medication; and
  • The medication can be administered, and it is logistically feasible to make it available, outside of the clinical trial setting.

The existence of an expanded access protocol does not guarantee any individual patient access to any specific experimental drug and, at this time, participation in one of our clinical trials is generally the most appropriate way to access our investigational drugs. If you have questions about an Ionis-sponsored clinical trial or our expanded access protocol, please speak with your physician or contact a study site listed on

Effecting change through community

In our pursuit to create a brighter future for patients, we must partner and collaborate to change the healthcare landscape, increase disease awareness and drive innovation.

Once in a lifetime breakthroughs again and again

Meet Sonia and Eric. When genetic testing revealed that Sonia would ultimately suffer from the same fatal prion disease as her mother, Sonia and her husband, Eric, refused to sit back and let the disease ravage their family.