Offering children with spinal muscular atrophy (SMA) type 1 the opportunity to move around on their own using motorized,…
Articles by Margarida Maia, PhD
After Spinraza (nusinersen) treatment for slightly longer than one year, school-aged children with spinal muscular atrophy (SMA) showed…
Gene therapy GC101 was found to rescue disease manifestations in a mouse model of spinal muscular atrophy (SMA) type…
It can be difficult for genetic counselors in the U.S. and Canada to explain indeterminate results of prenatal screening for…
Starting Evrysdi (risdiplam) very early — just 12 days after his birth and before his symptoms appeared — was…
Treatment with Spinraza (nusinersen) or Evrysdi (risdiplam) may help preserve upper limb motor function in children and young…
Testing patient DNA with advanced methods like HapSMA — which can “[analyze] … SMN and its surrounding genes” — can…
Children living with spinal muscular atrophy (SMA) in the U.K. can switch treatments based on practical needs and preferences…
The parts of cells that produce energy, called mitochondria, in the skeletal muscles involved in voluntary movements don’t work properly…
Spinraza (nusinersen) treatment may help restore balance to the levels of some lipids, fat-like molecules that are lower in…