Actor Colin Farrell recently disclosed that his son, James, has Angelman syndrome, one of the driving factors behind Farrell's support for finding a cure for Angelman syndrome and for launching the ...
Mothers are often encouraged to trust their instincts, especially regarding their children's health. But for Tiffany Young, getting others to take her concerns seriously proved challenging when her ...
In a discovery that opens the door to a less invasive way of treating some serious disorders before birth, UC San Francisco scientists have found that delivering medicine through amniotic fluid is as ...
Colin Farrell says his personal life has inspired him to launch a new foundation aimed at helping people with intellectual disabilities. The actor, 48, recently launched the Colin Farrell Foundation, ...
A rare genetic condition leads to developmental difficulties, happy disposition. Oct. 22, 2008— -- Six-year-old Ryan Ravellette is a happy and sociable child; he has a smile that can light up a ...
Affected populations: The disorder is believed to affect somewhere between 1 in 12,000 and 1 in 24,000 people, although these figures may be underestimated. Many cases of Angelman syndrome can go ...
Ultragenyx (RARE) Pharmaceutical announced that the first patient has been dosed in the pivotal Phase 3 Aspire study, NCT06617429, evaluating the efficacy and safety of GTX-102, its investigational ...
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 ...
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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NOVATO, Calif., Dec. 19, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (RARE), today announced that the first patient has been dosed in the pivotal Phase 3 Aspire study (NCT06617429) ...
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