Elevated GFAP Levels Linked to More Motor Impairment in SMA
Higher levels of the glial fibrillary acidic protein (GFAP) in the fluid around the brain and spinal cord are associated…
Articles by Marisa Wexler, MS
Newborn Screening Allows Timely SMA Treatment of Baby in Japan
In the first year of a newborn screening program for spinal muscular atrophy (SMA) in Japan, more than 10,000…
Compared to No Treatment for SMA Type 1, Outcomes Better With Evrysdi
Compared to what would be expected without treatment, the approved oral therapy Evrysdi (risdiplam) led to marked benefits in…
Moxifloxacin Boosts SMN Levels, Lifespan in SMA Cell, Mouse Models
Moxifloxacin, an antibiotic with a well-established safety profile, boosted production of the SMN protein in cellular models of spinal…
Spinraza Treatment May Help to Improve Arm Function in Adults
Among adults with spinal muscular atrophy (SMA) being treated with Spinraza (nusinersen), a decrease in levels of the…
Molecular Feedback Loop May Explain Motor Neuron Damage
The RNA-binding protein Mid1 increases levels of the mutated androgen receptor (AR) that drives nerve cell degeneration in spinal…
No New Issues for Evrysdi Reported in Compassionate Use Program
No new safety signals related to Evrysdi (risdiplam) were documented in a compassionate use program in Germany where more…
Spinraza Improves, Stabilizes SMA-related Motor Function: Study
Spinraza (nusinersen) treatment was generally well tolerated and led to improved or stabilized motor function for nearly 300 children…
Neurologists OK With Ambiguity More Likely to Pick Suboptimal Therapies
Pediatric neurologists who are more comfortable with ambiguity are more likely to make suboptimal treatment decisions for their patients with…
Medical Costs Lower if Treatment Is Started Before Symptoms: Study
The total medical costs associated with spinal muscular atrophy (SMA) are lower for patients diagnosed at birth via newborn…