n-Lorem Foundation and Ultragenyx United in Efforts to Help Nano-rare Patients

n-Lorem, a nonprofit foundation, announced today that Ultragenyx has continued its partnership with n-Lorem and provided a second donation to support n-Lorem's efforts to discover, develop and provide experimental antisense oligonucleotide (ASO) medicines for nano-rare patients (1 to 30 patients worldwide) for free, for life.

"Our patients need help today. Most are very sick and in desperate need for treatment. On behalf of our patients, I thank Ultragenyx and Dr. Emil Kakkis for their continued support, which translates directly into benefit today for our patients," said Stanley T. Crooke, M.D., Ph.D., Founder, CEO and Chairman of n-Lorem Foundation. "The challenges presented by nano-rare patients are far too complex for n-Lorem to solve alone. We have created a broad and effective network of partners that are the best of the best in the industry. Through their support and expertise, we can and are making a difference in the lives of nano-rare patients."

"At Ultragenyx, our mission is to treat as many individuals with rare disease as possible through a therapeutic development and commercial model that minimizes time and cost. Partnering with n-Lorem allows Ultragenyx to also make an impact on diseases where a commercial model would never be feasible," said Emil D. Kakkis, M.D., Ph.D., CEO and President of Ultragenyx Pharmaceutical. "We share Stan's urgency to help patients today and are honored to be a part of this important initiative by supporting n-Lorem's effort to reach patients who have limited to no therapeutic options."

Don't miss Drs. Crooke and Kakkis discussing the evolving role of patient advocacy this fall in n-Lorem's PEP podcast. As part of its mission to improve the lives of patients, n-Lorem launched an original podcast, the ?Patient Empowerment Program' (PEP). This podcast series consists of interviews and lectures that will provide listeners the tools to understand complex drug discovery and development and create a community for nano-rare patients and their families to connect and share their stories. Listen now to the first five episodes.

Learn more about n-Lorem's mission at www.nlorem.org, and please consider giving to n-Lorem to bring hope, possibility and treatment options to these needy patients and families.

• Subscribe: n-Lorem Patient Empowerment Program Podcast Series
• Watch: n-Lorem Foundation Delivers Hope and Treatment for Patient with Nano-Rare Disease
• Watch: n-Lorem Foundation: Creating a Better Future for Nano-Rare, One Patient at a Time

About n-Lorem

n-Lorem Foundation is a non-profit organization established to apply the efficiency, versatility and specificity of antisense technology to charitably provide experimental antisense oligonucleotide (ASO) medicines to treat nano-rare patients diagnosed with diseases that are the result of a single genetic defect unique to only one or very few individuals. Nano-rare patients describe a very small group of patients (1-30 worldwide) who, because of their small numbers, have few if any treatment options. n-Lorem Foundation was created to provide hope to these nano-rare patients by developing individualized ASO medicines, which are short strands of modified DNA that can specifically target the transcripts of a defective gene to correct the abnormality. The advantage of experimental ASO medicines is that they can be developed rapidly, inexpensively and are highly specific. To date, n-Lorem has assisted in the development and treatment of 14 nano-rare patients and received over 140 applications for treatment with more than 60 nano-rare patients approved. n-Lorem was founded by Stanley T. Crooke, M.D., Ph.D., former chairman and CEO of Ionis Pharmaceuticals, who founded Ionis Pharmaceuticals in 1989 and, through his vision and leadership, established the company as the leader in RNA-targeted therapeutics. Follow us on Twitter, Facebook, LinkedIn, YouTube and Instagram.