Dec 11, 2024 · Zolgensma (onasemnogene abeparvovec-xioi) and Spinraza (nusinersen sodium) are both treatments for people with spinal muscular atrophy (SMA). SMA is a type of motor neuron disease.

Efficacy of Spinraza (Nusinersen) in Treating Spinal Muscular Atrophy. Spinraza (Nusinersen) is an FDA-approved medication specifically designed to treat Spinal Muscular Atrophy (SMA), a rare genetic disorder characterized by loss of motor neurons, leading to …

Zolgensma is a gene therapy administered in a single intravenous dose that uses the adeno-associated virus serotype 9 vector (AAV9) to deliver a copy of the SMN1 gene to replace the native defective or absent gene.

Feb 22, 2019 · Evidence provides high certainty that both Spinraza and Zolgensma provide a substantial net health benefit compared to prior standard care in patients with infantile-onset (Type I) SMA.

Jan 30, 2022 · Researchers compiled data from studies of oral Evrysdi (risdiplam), Spinraza (nusinersen), and Zolgensma (onasemnogene abeparvovec), finding evidence that risdiplam may be a favorable...

Currently, zolgensma and nusinersen are the only two FDA-approved direct treatments for SMA. 24 Early detection and diagnosis of SMA are critical in preventing irreversible loss of motor function and muscular atrophy.

Apr 4, 2023 · Spinraza is a type of medication called an antisense oligonucleotide. It is used to treat children and adults with spinal muscular atrophy (SMA), a type of motor neuron disease. It was the first drug to be approved by the US Food and Drug Administration (FDA) for …

Apr 23, 2021 · Zolgensma may result in faster and greater functional improvements than Spinraza (nusinersen) in infants with spinal muscular atrophy (SMA) treated up to 2 years of age, according to a survey of 22 health providers in the U.S.

Dec 30, 2024 · Zolgensma (onasemnogene abeparvovec-xioi) and Spinraza (nusinersen sodium) are both treatments for people with spinal muscular atrophy (SMA). There are a number of key differences between the two treatments, including: Which patients with SMA they are approved for use in; How often they are administered; The type of treatments they are; How ...