MONTROSE, Mich. (WJRT) - A Mid-Michigan family is raising money to help their 6-year-old boy with a rare genetic disorder. After spending a few months looking for action figures and fun items, Kristin ...

KANSAS CITY, Mo. (KCTV) - Four-year-old Maddie loves cheese and fruit snack gummies, being right in the middle of the action, and playing with her twin sister, Kellie, and older brother, Colin. When ...

The Pinball Lounge at 78th Street Sudios offers an ideal setting for this unique fundraiser. The event runs from 4-6 pm, providing families with an accessible Sunday afternoon activity. True to its ...

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 ...

Angelman syndrome is a rare genetic disorder that impacts brain development, speech, movement, and learning.

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 ...

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 ...

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 ...

MIDDLETON, Mass., Sept. 22, 2025 /PRNewswire/ -- MavriX Bio, a clinical-stage biotechnology company focused on the development of transformative genetic therapies for Angelman syndrome (AS), today ...

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) ...