Falling levels of 4 proteins in CSF may predict response to Spinraza
A drop in the levels of four proteins in the cerebrospinal fluid (CSF) may be a marker of response to…
Articles by Patricia Inacio, PhD
Zolgensma safe, most effective when given to newborns: Real-world study
The one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) safely and effectively treats infants with spinal muscular atrophy (SMA), especially…
Spinraza benefits SMA children not helped by Zolgensma: Interim data
Most of the infants and children with spinal muscular atrophy (SMA) in the RESPOND clinical trial who responded poorly…
Best outcomes seen in real life with Zolgensma when given early: Study
In real-world settings, the one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) is well tolerated and improves motor function outcomes in…
Enzyme plays important role in protecting motor neurons: Cell study
The loss of SMN protein in spinal muscular atrophy (SMA) may lead to disease features by causing a deficiency…
Neurofilament levels may help diagnose adult-onset SMA vs ALS
The amount of neurofilament proteins found in the cerebrospinal fluid, the liquid surrounding the brain and spinal cord, may help…
Spinraza Doses Largely Given as Advised, Real-world US Data Show
People with spinal muscular atrophy (SMA) being treated with Spinraza (nusinersen) received most of their injections at recommended…
Teen with Rare SMA Form Named MDA National Ambassador for 2023
Leah Zelaya, a 15-year-old with a rare form of spinal muscular atrophy (SMA), was chosen by the Muscular Dystrophy…
SMA Youth Encouraged to Exercise With Interactive Video Game
Raft Digital Therapeutics has launched a demo version of Cloud Bazaar, a therapeutic video game that promotes physical activity and exercise…
EONIS Approved in US for Newborn Screening of SMA, SCID
PerkinElmer‘s EONIS system has been approved in the U.S. to be used by certified laboratories to simultaneously test newborns…