News

Spinraza-Zolgensma Combination Well-tolerated in Children with SMA Type 1, Study Shows

Combining Spinraza (nusinersen) with the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) is generally well-tolerated and sustains motor improvements in children with spinal muscular atrophy (SMA) type 1, according to a case series study. The data, which included children treated with Zolgensma at older ages than those reported in clinical trials, suggested…

Edema Reduction Massage In SMA Patients

As we’ve discussed in previous articles, most patients with SMA sit in a wheelchair all day, therefore increasing the likelihood of experiencing increased swelling in their feet and ankles. For able-bodied individuals, when they walk, their legs are in a gravity dependent position, and there is a natural…

Evrysdi for SMA Under Accelerated Review in Europe

The European Medicines Agency (EMA) has accepted and is now reviewing Roche’s marketing authorization application (MAA) requesting approval of Evrysdi (risdiplam) for treating spinal muscular atrophy (SMA). Given that Evrysdi previously received the EMA’s priority medicines designation for SMA, enabling accelerated assessment of its application, a decision may be…