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They created a drug for Susannah. What about millions of others?

Susannah Rosen, 8, spent much of her childhood in hospitals in New York City as doctors documented the gradual loss of her ability to stand, walk and see. ....

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They Created a Drug for Susannah. What About Millions of Other Patients?

Scientists have made rapid progress in customizing drugs for ultrarare diseases. The hard part now is making such treatments on a large scale. ....

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n-Lorem Makes Ultrarare Disease Treatment Personal

n-Lorem Makes Ultrarare Disease Treatment Personal
Joe Gleeson, MD
n-Lorem Makes Ultrarare Disease Treatment Personal
The nonprofit s founder pauses briefly at their one-year anniversary to reflect on hyper-personalized medicine without taking his eyes off the future.
April 15, 2021
Retirement typically means more time for traveling, hobbies, and relaxation. For Stan Crooke, MD, PhD, it means something very different. Crooke, the founder and executive chairman of Ionis Pharmaceuticals, having served as CEO for more than 30 years, will officially retire this year. Instead of hitting the golf course, he is throwing himself into the foundation he and his wife, Rosanne Crooke, PhD, started just over one year ago the n-Lorem foundation. The foundation’s goal is to provide free, individualized, antisense oligonucleotide (ASO) treatments to patients living with ultrarare (1 to 30 patients worldwide) diseases for life. The funding for n-Lorem comes from the Crookes ....

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Stanley T. Crooke, Ph.D., M.D, to retire from Ionis to focus on his scientific interests and n-Lorem Foundation

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CARLSBAD, Calif., Dec. 17, 2020 /PRNewswire/ Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapeutics, announced today that its founder and executive chairman Stanley T. Crooke, M.D., Ph.D., will retire from Ionis and its board of directors effective June 2021. Dr. Crooke is stepping down so that he may focus on his scientific interests and the nonprofit organization he founded, the n-Lorem Foundation. Dr. Crooke will continue to serve as a scientific advisor to Ionis including providing advice regarding Ionis research and development programs and guiding the core antisense research group he founded at Ionis. ....

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