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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.

New yorkUnited statesUnited kingdomColumbia universityLa jollaDavid coreyStanley crookeJulia vitarelloTimothy yuJennifer bainRosanne crookeSusannah rosenWendy chungJeff miltonSally jacksonMila makovec

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.

New yorkUnited statesColumbia universityUnited kingdomDavid coreyStanley crookeJulia vitarelloTimothy yuJennifer bainRosanne crookeSusannah rosenWendy chungJeff miltonSally jacksonMila makovecIssi rozen

Gene Treatment for Rare Epilepsy Causes Brain Side Effect in 2 Children

The side effect, a buildup of fluid in the brain, led to the death of one of the children and presents a grave setback for a class of personalized medicine.

United statesUniversity of massachusettsSeth greenblottTimothy yuAlexandra schenkelValeria schenkelLiza marie johnsonMila makovecLucy greenblottArthur kriegJude children research hospitalDrug administrationValeria associationTherapeutics instituteAmerican neurological associationBoston children

San Diego scientists race to treat rare genetic diseases - The San Diego Union-Tribune

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.

San diegoUnited statesSan franciscoMary cunninghamLisa hornakBob cunninghamIonis pharmacueticalsConnor dalbyLorem joseph ochabaLos angelesJoseph gleesonGemma bachmannKelley dalbyAmy danStanley crookeJoseph ochaba

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, Ionis, Biogen, a

United statesSan diegoStan crookeJulia vitarelloJoe gleesonRogcon biosciencesTimothy yuRosanne crookeNeil shneiderMila makovecKelley dalbyConnor dalby joe gleesonColumbia college of physiciansIonis pharmaceuticalsBoston childrenColumbia college