NGNE to update on a potential registrational phase 3 study, using NGN-401, for the treatment of patients with Rett Syndrome.

Acadia Pharmaceuticals Inc. today announced that the journal Med published results from the open-label study, DAFFODILtm, evaluating the safety, tolerability and exploratory efficacy of DAYBUE® ...

Neurotech International has received positive feedback from the European Medicines Agency (EMA) on the company’s Orphan ...

Ladies and gentlemen, good morning and welcome to the Taysha Gene Therapies full year 2024 conference call. (Operator Instructions). As a reminder, this conference is being recorded. It is now my ...

Ladies and gentlemen, good morning and welcome to the Taysha Gene Therapies Full Year 2024 Conference Call. At this time all participants are in listen-only mode. A brief question-and-answer session ...

High dose and low dose of TSHA-102 continue to be generally well tolerated with no treatment-related SAEs or DLTs in all pediatric, adolescent and adult patients treated (high dose, n=6; low dose, n=4 ...

High dose and low dose of TSHA-102 continue to be generally well tolerated with no treatment-related SAEs or DLTs in all pediatric, adolescent and adult patients treated (high dose, n=6; low dose, n=4 ...

Scientists have identified how Rett syndrome progresses at the molecular level. The loss of MeCP2 in adult brains triggers rapid gene dysregulation, independent of developmental factors. These ...

Jace, 6, was born with a rare and aggressive eye condition An experimental trial of gene therapy has helped four toddlers - born with one of the most severe forms of childhood blindness - gain ...

Rett syndrome is a rare genetic disease affecting 1 in 10,000 newborn girls, characterized by sudden regression around 1 year of age, with loss of acquired language and motor skills and leading to ...

Scientists suggest genetics played a big role – and they say the evolution of this singular ability was key to our survival. A new study links a particular gene to the ancient origins of spoken ...

A new study suggests that the NOVA1 gene may have been a key player in the evolution of human language. By Carl Zimmer Scientists have long struggled to understand how human language evolved.