In the time since the Novartis gene therapy Zolgensma was approved for babies with spinal muscular atrophy, other treatments ...

Investigators advised clinicians of the potential for dystrophies that mimic age-related macular degeneration and other ...

Spinal muscular atrophy type 1 is a genetic disorder that typically appears within the first six months of life, causing ...

Intrathecal onasemnogene abeparvovec (OAV101 IT), an investigational, one-time gene replacement therapy, demonstrated a ...

Delivery of onasemnogene abeparvovec into the intrathecal space was safe and effective for children with spinal muscular ...