England’s National Institute for Health and Care Excellence (NICE) issued its final Zolgensma’s appraisal, recommending that the gene therapy be covered by the country’s national health service (NHS) and provided at low or no cost to eligible spinal muscular atrophy (SMA) patients. The positive recommendation is consistent with England NHS’…
The initial position of England’s National Institute for Health and Care Excellence (NICE) is to recommend against adding Roche’s Evrysdi (risdiplam) to the list of medications available to spinal muscular atrophy (SMA) patients through the country’s national health service (NHS). Lack of evidence of Evrysdi’s efficacy in babies genetically diagnosed…
Blood levels of a stress-induced protein are significantly higher in young spinal muscular atrophy (SMA) patients and associate with levels of a nerve cell damage biomarker, a study shows. These findings from untreated SMA patients suggest that this protein, called heat shock 70kDa protein 7 (HSP70B), may be a…
The number of copies of the “backup” SMN2 gene and a rare genetic variant in this gene influence disease severity in people with spinal muscular atrophy (SMA), according to data from more than 2,000 people involved in a free SMA genetic testing program. Launched in 2018 by…
Spinal muscular atrophy (SMA) patients may at a fourfold greater risk of hydrocephalus, or abnormal accumulation of cerebrospinal fluid (CSF) in the brain, than their healthy peers, a large U.S. health record-based study suggests. Data also showed that patients younger than 2 years, those most likely to have more severe…
Apitegromab, Scholar Rock’s muscle-directed therapy for spinal muscular atrophy (SMA), was generally safe and showed dose-dependent accumulation and clearance of the inactive form of myostatin, a muscle growth suppressor, in healthy volunteers, according to data from a Phase 1 clinical trial. These findings, published in the journal Advanced…
The blood levels of neurofilaments — a marker of nerve cell damage — may serve as a disease onset and treatment response biomarker in infants with spinal muscular atrophy (SMA) type 1, but not in older patients with milder disease forms, a study suggests. These findings add to previous studies…
Novartis Gene Therapies has added thrombotic microangiopathy (TMA) — a rare condition characterized by blood abnormalities — to the list of safety concerns with use of Zolgensma in children with spinal muscular atrophy (SMA). This decision was driven by reports of six confirmed cases of TMA within one to two…
Sex and the number of copies of the “backup” SMN2 gene influence both the age of symptom onset and the risk of losing the ability to walk in people with spinal muscular atrophy (SMA) type 3, according to a Polish registry study. The data also confirmed the notion…
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. Given the survey’s limitations, such as its small sample and subjective…
