n-Lorem, a nonprofit foundation, announced 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”
“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.