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® ...

Biomed Industries, Inc. Biomed pipeline Biomed Industries, Inc. to Announce Phase 2/3 Results of NA-921 for the Treatment of Rett Sy ...

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 ...

Gene delivery is a process by which foreign DNA is transferred to host cells for applications such as genetic research or gene therapy. Gene delivery methods can be mechanical (e.g. microinjection ...

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 ...

Having two copies of the MTHFR gene mutation may raise your risk for certain health conditions, though more research is needed. You may have the mutation and not find out unless you undergo ...

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 ...

Pfizer’s decision to halt further marketing of Beqvez is further sign of the sparse patient interest in gene therapies for the bleeding condition. Rapid scientific advances have put the gene therapy ...