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) ...

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 ...

Colin Farrell has opened up on his difficult decision to place his disabled son in long-term care. James was born to Colin and ex-partner US model Kim Bordenave. The youngster was diagnosed with the ...

Though their bright smiles and arm-flapping exuberance suggests otherwise, victims of Angelmans Syndrome is so serious one South Side couple has started a non-profit to teach others about the rare ...

Company expects to complete Phase 3 Aspire study in the second half of 2026 Aurora study of GTX-102 in additional ages and genotypes on track to initiate in the second half of 2025 “The accelerated ...