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Carlsbad-based n-lorem develops specialized medicine for patients like 12-year-old Connor Dalby, a Carlsbad boy who could be the only person in the world living. ....
San Diego scientists race to treat rare genetic diseases - The San Diego Union-Tribune sandiegouniontribune.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from sandiegouniontribune.com Daily Mail and Mail on Sunday newspapers.
Empowering the Fight Against Rare Diseases One Disease at A Time clinicalomics.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from clinicalomics.com Daily Mail and Mail on Sunday newspapers.
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 ....
Share this article SAN DIEGO, Jan. 26, 2021 /PRNewswire/ n-Lorem Foundation, the first and only nonprofit organization whose mission is to create experimental antisense oligonucleotide (ASO) treatments for patients who have diseases caused by extremely rare mutations (1-30 patients worldwide) for free, for life, celebrates its first anniversary and the extraordinary progress made in 2020 toward its mission of offering immediate hope and rapid treatment to the millions of patients affected by ultra-rare mutations. In its first 12 months of operation, n-Lorem Foundation received applications to treat 50 patients, is proceeding to treat nearly 20 of those patients and helped two clinical investigators obtain experimental ASO treatments for nine patients – greatly exceeding applicant and acceptance rate expectations. Leveraging the powerful ASO technology developed at Ionis Pharmaceuticals, the foundation has established quality processes so that only appropriate pat ....