A recent review provides insight into the clinical progression of treatment-naive patients with spinal muscular atrophy type 3 who have lost ambulation. Patients with spinal muscular atrophy (SMA) ...

"The availability of disease-modifying therapies for patients with other types of SMA suggests that these key disease characteristics in SMA 3 patients should be amenable to therapy." — Barry J. Byrne ...

The new report is based on real-world data from a patient registry and shows some patients even regain motor function under treatment with nusinersen for spinal muscular atrophy (SMA). A new ...

Determining which patients should receive the treatment will still require individual risk-benefit assessments, experts say.

In two clinical trials, Evrysdi improved motor function in people living with SMA over a broad spectrum of ages and levels of disease severity, including Types 1, 2, and 3 SMA Evrysdi helped infants ...

man MS wheelchair Risdiplam is an investigational, orally administered liquid survival motor neuron-2 (SMN2) splicing modifier designed to increase and sustain SMN protein levels throughout the ...

** RULM is a scale designed to assess upper limb movement in people with SMA. It can capture progressive muscle weakness across the spectrum of the disease, reflective of the SUNFISH Part 2 study ...