Phase 1/2 data show improvements across all domains and confirm that Phase 3 Aspire study is amply powered to establish ...

Angelman syndrome is caused by several genetic mutations, but the most significant is loss of function in the maternally inherited form of the UBE3A gene, which leads to complications in the ...

The global Phase 3 Aspire study will enroll approximately 120 patients with Angelman syndrome with a genetically confirmed diagnosis of full maternal UBE3A gene deletion and will include a 48-week ...

Ultragenyx Pharmaceutical (NASDAQ:RARE) announced Saturday that GTX-102, its candidate for the rare neuro-genetic disorder Angelman syndrome, improved patient outcomes across multiple domains in a ...

TSHA-106 for the treatment of angelman syndrome; TSHA-114 for the treatment of fragile X syndrome; and TSHA-101 for the treatment of GM2 gangliosidosis. Receive News & Ratings for Taysha Gene ...

TSHA-106 for the treatment of angelman syndrome; TSHA-114 for the treatment of fragile X syndrome; and TSHA-101 for the treatment of GM2 gangliosidosis. Receive News & Ratings for Taysha Gene ...

Another promising program is GTX-102 for Angelman Syndrome, which is another genetic disease that impacts roughly 60K ...

(RARE) announced Phase 1/2 data in support of the Phase 3 Aspire study for GTX-102, its investigational antisense oligonucleotide for Angelman syndrome. The Phase 1/2 data showed improvements ...

Zolgensma gene therapy is the most effective in infants with SMA who are presymptomatic and 6 weeks old or younger, according ...

June 13, 2024 — A viral gene therapy has reversed some brain abnormalities in infant mice with FOXG1 syndrome, a significant step toward one day treating children with this severe neurodevelopmental ...