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n-Lorem Foundation and Ultragenyx united in their efforts to help nano-rare patients | Company


n-Lorem, a nonprofit foundation, today announced that Ultragenyx has continued its partnership with n-Lorem and made a second donation to support n-Lorem’s efforts to discover, develop and deliver investigational drugs. based Antisense Oligonucleotides (ASO) 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 of care. On behalf of our patients, I thank Ultragenyx and Dr. Emil Kakkis for their continued support, which translates directly into benefits for our patients today,” said Stanley T. Crooke, MD, Ph.D., Founder, CEO and President 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 large and efficient network of partners who are the best of the best in the industry. With their support and expertise, we can and do make a difference in the lives of nano-rare patients. »

“At Ultragenyx, our mission is to treat as many people with rare diseases as possible through a therapeutic and commercial development model that minimizes time and cost. Partnering with n-Lorem enables Ultragenyx to also impact diseases for which a commercial model would never be feasible,” said Emil D. Kakkis, MD, Ph.D., CEO and President of Ultragenyx Pharmaceutical . “We share Stan’s urgency to help patients today and are honored to be part of this important initiative by supporting n-Lorem’s efforts to reach patients who have few or no treatment options.

Don’t miss Drs. Crooke and Kakkis discuss the changing role of patient advocacy this fall on 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 with 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, and please consider donating to n-Lorem to bring hope, possibility, and treatment options to these patients and families in need.

  • Subscribe: n-Lorem Patient Empowerment Program Podcast Series
  • look: The n-Lorem Foundation brings hope and treatment to a patient with a nano-rare disease
  • look: n-Lorem Foundation :Creating a better future for the nano-rare, one patient at a time

About n-Lorem

The n-Lorem Foundation is a non-profit organization created to apply the efficacy, versatility, and specificity of antisense technology to provide charitable investigational antisense oligonucleotide (ASO) drugs to treat diagnosed nano-rare patients. With diseases which are the result of a single genetic defect unique to a single or very few individuals. Nano-rare patients describe a very small group of patients (1 in 30 worldwide) who, due to their small numbers, have few or no treatment options. The n-Lorem Foundation was created to bring hope to these nano-rare patients by developing individualized ASO drugs, which are short strands of modified DNA that can specifically target the transcripts of a faulty gene to correct the ‘anomaly. The advantage of ASO investigational drugs is that they can be developed quickly, inexpensively and are highly specific. To date, n-Lorem has assisted in the development and treatment of 14 nano-rare patients and has received over 140 treatment requests with over 60 nano-rare patients approved. n-Lorem was founded by Stanley T. Crooke, MD, Ph.D., former President and CEO of Ionis Pharmaceuticals, who founded Ionis Pharmaceuticals in 1989 and, through his vision and leadership, has makes the company the leader in targeted RNA. therapeutic. follow us on TwitterFacebook, LinkedIn, YouTube and Instagram.

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CONTACT: n-Lorem Contact:

Amy Williford, Ph.D.

Senior Director of Communications and Donor Relations

[email protected]



SOURCE: n-Lorem Foundation

Copyright BusinessWire 2022.

PUBLISHED: 06/20/2022 09:05 AM/DISC: 06/20/2022 09:06 AM

Copyright BusinessWire 2022.