NEW YORK, Sept. 16, 2025 /PRNewswire/ -- Myrtelle Inc. ("Myrtelle" or the "Company), a pioneering clinical-stage gene therapy company developing transformative treatments for neurodegenerative ...

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.

- 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 ...

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 ...

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 ...

- 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 ...

- 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 ...

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 ...

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 ...

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