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Ionis' (IONS) Angelman Syndrome Drug Gets FDA's Orphan Tag
Zacks via Yahoo Finance· 2 years agoThe FDA grants orphan drug and rare pediatric disease designations to Ionis' (IONS) ION582 for...
Ultragenyx (RARE) Provides Update From Angelman Syndrome Study
Zacks via Yahoo Finance· 2 years agoUltragenyx (RARE) and GeneTx Biotherapeutics provide an update and positive interim data from the...
Colorado Springs to host ASF Walk
KXRM Colorado Springs via Yahoo News· 3 weeks ago(COLORADO SPRINGS) — In celebration of 25 years, the Angelman Syndrome Foundation (ASF) will host its yearly walk the weekend of Saturday, May 18....
‘Meet Angelman syndrome’: A Rosemount toddler’s diagnosis spurs family to work for cure
Pioneer Press, St. Paul, Minn. via Yahoo News· 1 year agoHenry Edberg dug through the play kitchen at his home in Rosemount before serving up the daily special to his older brother. “Vegetables are healthy, so I’m giving you the vegetables,” Henry ...
Ultragenyx (RARE) Meets Efficacy Goal in Neuro Disease Study
Zacks via Yahoo Finance· 1 month agoUltragenyx (RARE) reports positive interim data from the phase I/II study of GTX-102 for Angelman...
Harper's Hope: Webb continues to navigate rare disease diagnosis after 10 years
Messenger-Inquirer, Owensboro, Ky. via Yahoo News· 1 year agoMar. 7—Ten-year-old Harper Webb, a fourth-grader at Highland Elementary School, was diagnosed with Angelman Syndrome at 11 months old, and despite the diagnosis, she is a bright and kind-hearted ...
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) Q4 2022 Earnings Call Transcript
Insider Monkey via Yahoo Finance· 1 year agoUltragenyx Pharmaceutical Inc. (NASDAQ:RARE) Q4 2022 Earnings Call Transcript February 16, 2023...
I dreamed of the travel we'd do as a family of 4. Then came my youngest daughter's life-altering...
Business Insider via Yahoo News· 4 months agoAuthor and her eldest daughter on vacation at a hotel in Sydney.Tatyana Leonov Tatyana Leonov and...
Ultragenyx says Angelman therapy is working, but safety questions remain
BioPharma Dive via Yahoo Finance· 2 months agoTreatment appeared to result in functional and cognitive gains in people with the neurological...
How Angie Kim's immigrant struggles inspired a heart-wrenching thriller
LA Times via Yahoo News· 9 months agoAngie Kim's second novel, 'Happiness Falls,' pivots on a boy whose Angelman syndrome locks him in. For the author, it brought back memories of immigration.
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