Ionis Pharmaceuticals Supports Rare Disease Day

Feb 29, 2016, 07:00 ET from Ionis Pharmaceuticals, Inc.

CARLSBAD, Calif., Feb. 29, 2016 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) recognizes International Rare Disease Day and has partnered with the Amyloidosis Research Consortium (, Cure SMA (, and Myotonic Dystrophy Foundation ( to raise awareness of those living with rare diseases. As part of Ionis' initiative to recognize the value of bringing together researchers, patients, caregivers and advocacy groups, Ionis will be hosting a seminar for its employees.  Themed 'Patient Voice' this event emphasizes the crucial role patients, their families and caregivers play in raising disease awareness and instigating change to improve their lives and the lives of others. 

"We are proud to join forces with the patient community in celebration of Rare Disease Day. We stand in support of all those affected by rare diseases. We are aware of the significant, urgent need to bring new treatments to these patients," said Stan Crooke, M.D., Ph.D., chief executive officer and chairman at Ionis Pharmaceuticals. "At Ionis we are driven to create medicines with the potential to significantly impact the lives of patients. We have created one of the largest severe and rare disease drug pipelines in the industry with the hope that we can create better, healthier futures for patients with severe and rare diseases."

Rare Disease Day is an international campaign designed to focus attention on the impact of rare diseases. February 29, 2016, marks the ninth international Rare Disease Day. On and around this day hundreds of patient organizations from countries and regions all over the world will hold awareness-raising activities. For more information about Rare Disease Day, visit

ABOUT IONIS PHARMACEUTICALS, INC. Ionis is the leading company in RNA-targeted drug discovery and development focused on developing drugs for patients who have the highest unmet medical needs, such as those patients with severe and rare diseases. Using its proprietary antisense technology, Ionis has created a large pipeline of first-in-class and/or best-in-class drugs, with over a dozen drugs in mid- to late-stage development.  Drugs currently in Phase 3 development include volanesorsen, a drug Ionis is developing and plans to commercialize through its wholly owned subsidiary, Akcea Therapeutics, to treat patients with familial chylomicronemia syndrome and familial partial lipodystrophy; IONIS-TTRRx, a drug Ionis is developing with GSK to treat patients with all forms of TTR amyloidosis; and nusinersen, a drug Ionis is developing with Biogen to treat infants and children with spinal muscular atrophy. Ionis' patents provide strong and extensive protection for its drugs and technology. Additional information about Ionis is available at

IONIS' FORWARD-LOOKING STATEMENT This press release includes forward-looking statements regarding Ionis Pharmaceuticals' business and the therapeutic and commercial potential of Ionis' technologies and products in development, including nusinersen, IONIS-TTRRx and volanesorsen.  Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement.  Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs.  Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements.  Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis.  As a result, you are cautioned not to rely on these forward-looking statements.  These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended December 31, 2015, which is on file with the SEC.  Copies of this and other documents are available from the Company.

In December, 2015, the Company changed its name from Isis Pharmaceuticals, Inc. to Ionis Pharmaceuticals, Inc.  In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our," and "us" refers to Ionis Pharmaceuticals and its subsidiaries.

Ionis Pharmaceuticals™ is a trademark of Ionis Pharmaceuticals, Inc.  Akcea Therapeutics™ is a trademark of Ionis Pharmaceuticals, Inc. 

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SOURCE Ionis Pharmaceuticals, Inc.