Neurogene has reported encouraging efficacy results with its gene therapy for rare genetic disorder Rett syndrome, but investors' concerns about safety seem to be weighing on its share price.

Neurogene has halted patient enrolment in the high-dose arm of its trial of Rett syndrome gene therapy, sparking another run on its shares. Last week, the company reported that NGN-401 had shown ...

Among individuals with Rett syndrome, treatment with trofinetide was linked to improvements in caregiver-reported outcomes by ...

Rett syndrome follows a unique temporal profile and is often characterized with stereotypic hand movements, behavioral issues and altered growth profiles. No treatment exists to cure this disease ...

Taysha's cash runway extends to Q4 2026, but likely capital raises could dilute shareholders, adding to downside risk. Click ...

Marvel Biosciences Corp. (TSXV: MRVL) (OTCQB: MBCOF), and its wholly owned subsidiary, Marvel Biotechnology Inc. (together ...

Concord High School is experiencing a duck invasion. The birds line the cafeteria coffee bar, rest on teachers’ and ...

Neurogene (NGNE) announced a peer-reviewed publication on its EXACT transgene regulation technology. The preclinical ...

Neuren Pharmaceuticals (ASX:NEU) and its US partner Acadia Pharmaceuticals have been blitzing it with its Rett syndrome drug ...