Business Wire

Myrtelle Announces Significant Reduction in N-Acetylaspartate (NAA), a Key Biomarker, in Patients Treated in Its Phase 1/2 Clinical Trial of the Investigational Gene Therapy ...

Analysis of Cerebral Spinal Fluid (CSF) from patients at pre-treatment baseline to post-treatment intervals up to 24 months demonstrates marked decrease in N-Acetylaspartate (NAA) Magnetic Resonance ...
Morningstar

Myrtelle Announces Nature Medicine Publication of Interim Results from Its Phase 1/2 Clinical Trial of Investigational Gene Therapy rAAV-Olig001-ASPA for Canavan Disease

Groundbreaking study demonstrates that targeted oligodendrocyte gene therapy leads to decreased N-acetylaspartate (NAA) levels and increased brain myelin volume, resulting in promising functional ...
Nasdaq

BridgeBio Shares Positive Data from High Dose Cohort of Phase 1/2 CANaspire Study of Gene Therapy BBP-812 for Canavan Disease at ESGCT 2024

- Continued, progressive improvement in motor function and achievement of motor milestones at 12-months post-treatment represents an important and statistically significant change, in contrast to the ...
News Medical

Preclinical MRI for neurological diseases with cellular and molecular imaging

Widespread neurological conditions, including Alzheimer’s disease, Parkinson’s disease, epilepsy, multiple sclerosis, stroke, and brain tumors, are among the top causes of disability and death around ...
Seeking Alpha

BridgeBio Receives FDA’s Regenerative Medicine Advanced Therapy (RMAT) Designation for BBP-812 Canavan Disease Gene Therapy Program

- Receipt of RMAT Designation is based on preliminary clinical evidence from the CANaspire Phase 1/2 clinical trial, which showed functional improvements in all dosed patients indicating that BBP-812 ...