"She is a little warrior, she is so strong. Within the last 12 months we found she has regressed even more. Isla is literally ...

At Neurogene, the developer of a gene therapy for Rett syndrome, expediency seems to be the priority, @adamfeuerstein writes.

Taysha Gene Therapies, Inc. is a clinical-stage biotechnology company focusing on adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system, with a ...

Neuren Pharmaceuticals (ASX:NEU), an Australian biotech focused on developing treatments for neurological disorders, has ...

High dose TSHA-102 was generally well tolerated with no SAEs or DLTs in two adolescent/adult patients and one pediatric ...

Coleen Rooney shares emotional details of her foster sister Rosie's life, calling her the family's "biggest inspiration" despite heartbreaking challenges.

New York, USA-based rare neurological disease drug developer Neurogene has announced positive interim clinical data in the first four participants in the low-dose cohort of its ongoing Phase I/II open ...

Biotech stock Neurogene collapsed Tuesday after a patient experienced a serious side effect in the company's Rett syndrome ...

Neurogene reports interim Phase 1/2 trial results for NGN-401 gene therapy in treating pediatric Rett syndrome, showing ...

Neurogene shares tumbled 35% last night after it disclosed that a pediatric patient with Rett syndrome experienced a severe ...

When looking at Neurogene’s stock performance year-to-date, it’s hard to ignore the impressive +269.09% increase. However, this recent downturn suggests that the market is responding to something ...

Analysts did not seem very concerned by the treatment-related serious adverse event, noting that NGN-401 was well-tolerated ...