Genetic engineering eases motor symptoms in SMA mice
Modifications that help certain genetic information travel to specific parts of nerve cells can ease motor symptoms and help normalize…
Articles by Lila Levinson, PhD
Specially designed back brace helps ease scoliosis in SMA children
Using a specially designed back brace in addition to taking part in physical therapy may work best to reduce…
Metric may predict response to SMA gene therapy Zolgensma
A technique to estimate the health of nerve cells that control movement may help predict how strongly some individuals will…
Biogen acquires Alcyone, device aimed at improving drug delivery
Biogen has acquired Alcyone Therapeutics and will continue to develop ThecaFlex DRx, a device intended to ease patient…
Blood test may help track response to SMA treatment Evrysdi
Measuring the levels of the survival motor neuron (SMN) protein in blood cells may help track spinal muscular atrophy (SMA)…
Man’s case shows how SMA muscle loss may lead to diabetes
A man with type 3 spinal muscular atrophy (SMA) and no history of diabetes developed diabetic ketoacidosis, a severe…
Study shows SMA infants with neutropenia can take Zolgensma
An infant with spinal muscular atrophy (SMA) type 1 and neutropenia was successfully treated with with Zolgensma (onasemnogene…
Majority of parents in UK support national SMA newborn screening
Most parents and potential parents in the U.K. support screening newborns for spinal muscular atrophy (SMA) as part of…
Brain fluid biomarkers may help predict Spinraza efficacy in SMA
Molecular markers in the liquid that surrounds the brain and spinal cord, called the cerebrospinal fluid (CSF), may help predict…
SMA patient impacts measured by new PROfuture questionnaire set
A group of questionnaires called PROfuture can meaningfully measure a variety of symptoms of spinal muscular atrophy (SMA) that…