Angelman syndrome is a rare, neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene. The maternal-specific inheritance pattern of Angelman syndrome is ...

The initial participant in the pivotal Phase 3 REVEAL trial, which is assessing ION582 for Angelman syndrome, a severe neurodevelopmental disease, has been dosed by Ionis Pharmaceuticals, Inc ...

Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the first participant has been dosed in the global Phase 3 REVEAL study, which is designed to evaluate the efficacy and safety of ...

Ionis has committed to a phase 3 programme for its antisense therapy for rare genetic disorder Angelman syndrome, just weeks after Biogen decided against exercising an option to license the drug.

Dead butt syndrome occurs when the gluteus medius stops firing due to unuse or sitting all day. Here are 5 exercises to help activate your glutes right now.

Phase 3 Aspire study enrollment on track to complete in 2025 Aurora study to evaluate GTX-102 in other Angelman syndrome genotypes and ages expected to initiate later this year NOVATO, Calif., June 27 ...

The proper treatment for dead butt syndrome will depend on how far it has progressed and on your physical activity goals. If you’re a runner trying to get back on track as soon as possible, you ...

Owing to its overwhelming predominance in females, Rett syndrome was regarded as a genetic disorder. However, its occurrence is sporadic, with a recurrence risk well below 0.5%.

Mammals are diploid creatures. Every cell in their body contains two sets of chromosomes, one from the mother and one from the father. Mouse embryos with only paternal genomes can not develop to term, ...