- 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 ...

Fern Kupfer of Ames is author of "Before and After Zachariah" and a contributor to the Iowa Writers Collaborative Substack. I got a call from a very nice woman who said she was in charge of unclaimed ...

- 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 ...

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 ...

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

GREENWOOD TODAY IS TO WITNESS A MIRACULOUS JOURNEY FROM BIRTH. NOAH’S STORY SEEMED LIKE ANY OTHER VERY NORMAL BIRTH, NORMAL BABY IN THE BEGINNING, LIKE SHE WAS JUST COMPLETELY DELIGHTFUL. BUT SOON ...

- 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 ...

The first peer-reviewed journal in the field of human gene therapy, providing all-inclusive coverage of the research, methods, and clinical developments that are driving today's explosion of gene ...