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…
Moxifloxacin, an antibiotic with a well-established safety profile, boosted production of the SMN protein in cellular models of spinal muscular atrophy (SMA) and it extended the lifespan and improved motor function in mice with the disease, a new study reports. “Preclinical data from patient-derived MNs [motor neurons] and a…
Among adults with spinal muscular atrophy (SMA) being treated with Spinraza (nusinersen), a decrease in levels of the inflammatory protein YKL-40 in the fluid around the brain is associated with improvements in arm and hand function, according to a new study. “We conclude that YKL-40 in CSF [cerebrospinal…
The RNA-binding protein Mid1 increases levels of the mutated androgen receptor (AR) that drives nerve cell degeneration in spinal and bulbar muscular atrophy (SBMA), a new study shows. Since Mid1 is expressed mainly in lower motor neurons, the nerve cells mainly affected in SBMA, the study’s researchers proposed that…
No new safety signals related to Evrysdi (risdiplam) were documented in a compassionate use program in Germany where more than 100 people with spinal muscular atrophy (SMA) were treated with the oral therapy prior to its approval last year, a new study reports. “We present the first real-world…
