Respiratory Weakness in SMA Most Pronounced in Childhood, Study Finds
Lung function in people with earlier-onset forms of spinal muscular atrophy (SMA) tends to decline gradually throughout childhood and…
Articles by Joana Carvalho, PhD
SBMA Seen to Affect Skeletal Muscles First, Not Motor Neurons
Although a disease of motor neurons, nerve cells of the spinal cord and brainstem, the earliest signs of spinal…
Interplay of Motor Neurons and Glial Cells at Root of SMA, Review Suggests
The interplay between different types of nerve cells, including motor neurons and glial cells, seems to be at…
Growth-friendly Spinal Implants May Help SMA Children Before Fusion Surgery
Spinal fusion surgery — a procedure to fuse two or more spine vertebrae — is more successful at reducing…
Delays in Diagnosing SMA, Especially Type 3, Still Evident, Study Finds
Delays in diagnosing spinal muscular atrophy are still evident, particularly for those with SMA type 3, despite recent changes…
First Patient Treated in Phase 2/3 Trial Assessing Higher Dose of Spinraza
The first patient has been dosed in the Phase 2/3 DEVOTE trial assessing the safety, tolerability, and efficacy of…
Motor Gains and Safety with Zolgensma’s IT Use Continue, STRONG Data Show
Young children given a single injection of Zolgensma into the spinal canal continue to show gains in motor function…
CHMP Recommends Conditional Approval of Zolgensma for SMA in Europe
The European Medicine Agency’s Committee for Medicinal Products for Human Use (CHMP), has recommended conditional approval of Zolgensma…
Zolgensma Shows Benefits and Durability Across Trials in Infants, Novartis Says
A single infusion of Zolgensma (onasemnogene abeparvovec-xioi) continues to lead to meaningful and sustained benefits in children with spinal…
SYT13 Gene Can Prolong Life in Mice With ALS, SMA, Study Shows
A gene called synaptotagmin 13 (SYT13) can prolong the lifespan and prevent the destruction of motor neurons in mouse…