Evrysdi safe for patients moving from other SMA therapies: Trial
Switching to oral Evrysdi (risdiplam) from another disease therapy is safe for spinal muscular atrophy (SMA) patients, with…
Articles by Lindsey Shapiro, PhD
Adherence to Evrysdi treatment found high in real-world analysis
Spinal muscular atrophy (SMA) patients in the U.S. exhibited high rates of adherence to their prescribed regimen of…
New lab test may help in studying effects of Spinraza treatment
Researchers have developed a new laboratory technique for measuring levels of Spinraza (nusinersen), an approved treatment for spinal muscular…
Abnormal immune system activation seen in SMA fly models
A range of genetic mutations associated with spinal muscular atrophy (SMA) were linked to abnormal activation of the immune…
More work needed to understand SMA fatigue mechanisms: Review
More high-quality studies are needed to understand the mechanisms of fatigue in spinal muscular atrophy (SMA) and to…
Stem cell-derived vesicles show promise for SMA in mouse model
Tiny carriers of cellular cargo called extracellular vesicles (EVs) — isolated from stem cells found in fat tissue — improved…
Troponin T protein elevated in SMA; might reflect muscle damage
The troponin T (TNT) protein — a marker of heart muscle damage — was elevated in the bloodstream of more…
SMA research offers clues into how splice-modifying therapies work
New research has identified mechanisms by which Evrysdi (risdiplam), an approved spinal muscular atrophy (SMA) therapy, has more…
New technique helps ease problem with swallowing in man with SMA
The practice of vacuum swallowing, a maneuver involving effort that’s intended to aid the movement of food through the throat,…
Learning rare lessons with Evrysdi compassionate use program: Report
Implementing the global compassionate use program (CUP) that enabled more spinal muscular atrophy (SMA) patients to access Evrysdi…