Actor Colin Farrell has opened up about the difficult decision to move his son James, who has Angelman syndrome, into a long-term care facility. In an interview this week the actor, 48, revealed he ...
NOVATO, Calif., Jan. 03, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious ...
Nancy Brady has been gratified to see the tool she and colleagues pioneered over a decade ago to measure the growth of infants' pre-speech communication skills translated into several languages and ...
NOVATO, Calif., Dec. 19, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), today announced that the first patient has been dosed in the pivotal Phase 3 Aspire study (NCT06617429) ...
A two-phase NIH grant will fund research into a new CRISPR-based gene therapy platform that will target genetic brain diseases like Angelman syndrome and H1-4 (HIST1H1E) syndrome. A roughly $40 ...
This post was updated on April 24, 2019 to incorporate feedback from parents. Names have been changed to protect anonymity. Is this child happy? Source: Joel Frohlich (AI generated with Midjourney) ...
Actor Colin Farrell has opened up about the difficult decision to move his son James, who has Angelman syndrome, into a long-term care facility. The Oscar-nominated star of The Banshees of Inisherin, ...
Ultragenyx Pharmaceutical Inc. announced the dosing of the first patient in its Phase 3 Aspire study, which is investigating the safety and efficacy of GTX-102, an investigational antisense ...
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