- 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 ...

The START pilot program aims to accelerate the development of novel drug and biological products for rare diseases rAAV-Olig001-ASPA was selected as one of a few CBER-regulated products based on ...

- 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 ...

- Pharmacodynamic data from the first three participants show sustained reductions in N-acetylaspartate (NAA) in the brain and urine, suggesting that the investigational therapy is producing ...

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

To continue reading this content, please enable JavaScript in your browser settings and refresh this page. Within the first few weeks after Lori and Lee Greenwood’s ...

Canavan disease is a rare, inherited leukodystrophy arising from mutations in the ASPA gene, which result in reduced activity of the enzyme aspartoacylase. This enzyme deficiency leads to the ...

NEW YORK, Oct. 6, 2025 /PRNewswire/ -- Myrtelle Inc. ("Myrtelle" or the "Company"), a gene therapy company pioneering transformative treatments for neurodegenerative diseases, today announced that Dr.

- Following treatment, the N-acetylaspartate (NAA) levels of CANaspire participants are consistent with levels seen in individuals with milder Canavan disease based on findings from the Company’s ...