News

Taysha Gene Therapies: Sell Rating On Uncertain Pipeline Prospects And Limited Opportunity
Taysha's cash runway extends to Q4 2026, but likely capital raises could dilute shareholders, adding to downside risk. Click ...
Healio8d
Trofinetide improves behavioral, physical outcomes in Rett syndrome at 9 months
Among individuals with Rett syndrome, treatment with trofinetide was linked to improvements in caregiver-reported outcomes by ...
pharmaphorum2d
Neurogene shares fall on Rett syndrome gene therapy data
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.
Marvel Biosciences to Give Oral Presentation at The Annual IRSF Rett Syndrome Scientific Meeting
Marvel Biosciences Corp. (TSXV: MRVL) (OTCQB: MBCOF), and its wholly owned subsidiary, Marvel Biotechnology Inc. (together ...
pharmaphorum7d
Neurogene falls again on Rett gene therapy side effects
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 ...
Dr Boreham’s Crucible: Drug-development unicorn Neuren banks on ‘cascade of long-term growth’
Neuren Pharmaceuticals (ASX:NEU) and its US partner Acadia Pharmaceuticals have been blitzing it with its Rett syndrome drug ...
Seeking Alpha1d
TSHA Taysha Gene Therapies, Inc.
TSHA-102 for the treatment of Rett syndrome; TSHA-121 for the treatment of CLN7 disease; TSHA-118 for the treatment of CLN1 disease; TSHA-105 for the treatment of SLC13A5 deficiency; TSHA-113 for ...
Neurogene announces peer-reviewed publication of EXACT transgene technology
Neurogene (NGNE) announced a peer-reviewed publication on its EXACT transgene regulation technology. The preclinical ...