Combining Evaluation Tools May Detect Early Signs of SMA: Study
Combining two clinical assessment tools helped recognize early neurological signs in infants with spinal muscular atrophy (SMA) who were…
Articles by Steve Bryson, PhD
MicroRNAs Can Predict Response to Spinraza, Study Suggests
MicroRNAs isolated from people with spinal muscular atrophy (SMA) types 2 and 3 before treatment predicted eventual responses to…
Disease Severity May Be Affected by SMN Protein in Rare SMA Cases
The stability and self-interacting ability of SMN, the protein that is abnormally low in people with spinal muscular atrophy…
Combining Therapies May Improve Efficacy in SMA: Mouse Model
A combined spinal muscular atrophy (SMA) therapy — one incorporating two treatments targeting different disease processes related to the…
Spinraza Most Effective in Younger Type 1 Children: Real-world Study
In a real-world study involving more than 40 Swiss patients with spinal muscular atrophy (SMA), Spinraza (nusinersen) was found to…
Same Mutation Causes SMABF2 in 2 Unrelated Infants, Case Study Says
The same mutation caused spinal muscular atrophy with congenital bone fractures-2 (SMABF2) in two unrelated infants of Roma descent,…
2 Spinraza Trials Now Enrolling, With ASCEND Testing Higher Dose
Children, teenagers, and adults with later-onset spinal muscular atrophy (SMA), previously treated with Evrysdi (risdiplam), can now enroll…
Using Ultrasound May Help in SBMA Diagnosis, Study Shows
Men with spinal and bulbar muscular atrophy (SBMA) were found to have abnormally small nerves in the limbs, especially…
Negotiations on Evrysdi Availability, Pricing in Canada Now Complete
Roche Canada has completed its negotiations with the pan-Canadian Pharmaceutical Alliance (pCPA) regarding the availability and pricing of…
UK Expands Referral Criteria for Zolgensma to Heavier Children
The U.K. has expanded the referral criteria for the approved gene therapy Zolgensma to include children with spinal…