Treatment with Evrysdi (risdiplam) increased levels of SMN protein and stabilized motor function in a Phase 2 clinical trial that enrolled spinal muscular atrophy (SMA) patients who had previously been on other therapies, two-year data show. “These important data demonstrate the safety and efficacy of Evrysdi in a…
The gene therapy Zolgensma (onasemnogene abeparvovec-xioi), approved in the U.S. for very young children with spinal muscular atrophy (SMA), potentially could work for adults with the rare genetic disorder, a new study suggests. The study reports that few adults with SMA were found to have antibodies against the…
Doctors need to be well-educated about options in the evolving treatment landscape to help patients with spinal muscular atrophy (SMA) make informed decisions, say neurologists in a panel discussion. The panel of four neurologists with experience in treating SMA patients was convened by Biogen, which markets…
Note: This story was updated Sept. 19, 2022, to correct that 78.9% of the survey respondents would choose an ASO or a small molecule therapy, such as Evrysdi (risdiplam), if they were approved therapies. Many members of the spinal muscular atrophy (SMA) community are interested in the…
In its final decision, the Canadian Agency for Drugs and Technologies in Health (CADTH) recommended against Spinraza (nusinersen) being reimbursed to treat adults with spinal muscular atrophy (SMA). The decision was met with dismay by the SMA community and Biogen, Spinraza’s manufacturer. “We are absolutely devastated for the…
Prescribing information for Zolgensma (onasemnogene abeparvovec-xioi), an approved gene therapy for spinal muscular atrophy (SMA), is being updated following the death of two patients who developed acute liver failure after treatment. Acute liver failure was known to be a potential severe side effect of Zolgensma, highlighted in a…
Children diagnosed with spinal muscular atrophy (SMA) are usually informed about the diagnosis by their parents, who frequently lack support and information about the disease, ultimately leading to poor communication that can be emotionally painful for everyone involved, according to a new study. “Poor communication has a double negative…
Higher levels of the glial fibrillary acidic protein (GFAP) in the fluid around the brain and spinal cord are associated with poorer motor function among people with spinal muscular atrophy (SMA), a new study reports. These results indicate that cells called astrocytes are more active in advanced SMA, according…
In the first year of a newborn screening program for spinal muscular atrophy (SMA) in Japan, more than 10,000 babies were screened and one tested positive for the disease, according to a new study. The baby with SMA was able to receive the gene therapy Zolgensma (onasemnogene abeparvovec-xioi)…
Compared to what would be expected without treatment, the approved oral therapy Evrysdi (risdiplam) led to marked benefits in survival and motor outcomes for babies with spinal muscular atrophy (SMA) type 1, according to a comparison of clinical trial data. “Untreated infants with Type 1 SMA … reached…
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