News

The newborn screening program at Cadham Provincial Laboratory in Manitoba, Canada, is expanding to include spinal muscular atrophy (SMA), according to an announcement from Audrey Gordon, the province’s health minister. “Early diagnosis is the key to improving outcomes for babies born with metabolic, endocrine or genetic disorders such as…

A new modeling study predicts that higher doses of Spinraza (nusinersen) may improve its effectiveness compared with the approved 12 mg dose. The study used data from two clinical trials that evaluated the disease-modifying therapy in infants with spinal muscular atrophy (SMA). “The [pharmalogical] model indicates that the…

Among adults with spinal muscular atrophy (SMA) type 3, those with greater motor impairment tend to score better on some cognitive measures, but worse on others, a new study reports. Among those with SMA, “patients with greater motor difficulties had lower performance in attention, but higher performance in measures…

Combining two clinical assessment tools helped recognize early neurological signs in infants with spinal muscular atrophy (SMA) who were identified through a newborn screening program, a study suggested. Even in the absence of obvious clinical signs, infants typically defined as presymptomatic may have minimal signs, such as poor muscle…

Infants with spinal muscular atrophy (SMA) who are being screened for Zolgensma may present with maternal antibodies against the therapy’s viral carrier — making them ineligible for the therapy — until the age of 8 months, a small study shows. This means that an infant first testing positive for…

The U.S. Food and Drug Administration (FDA) has extended its approval of Evrysdi (risdiplam) to treat babies with spinal muscular atrophy (SMA) who are younger than 2 months old. Evrysdi, an oral therapy that works by increasing the levels of the survival motor neuron (SMN) protein that is missing in…

MicroRNAs isolated from people with spinal muscular atrophy (SMA) types 2 and 3 before treatment predicted eventual responses to Spinraza (nusinersen) therapy, a study suggested. The study, “Muscle microRNAs in the cerebrospinal fluid predict clinical response to nusinersen therapy in type II and type III…

Development of an exoskeleton suit to help those living with progressive neuromuscular conditions that affect their upper-body movement will advance due to a £1.25 million (about $1.5 million) grant from the People’s Postcard Lottery in the United Kingdom. The “SMART Suit,” which targets people with disabilities such as…

A committee of the Canadian Agency for Drugs and Technologies in Health (CADTH) has issued a draft recommendation advising Spinraza (nusinersen) should not be reimbursed for the treatment of adults with spinal muscular atrophy (SMA).  CADTH is an independent, nonprofit organization that provides evidence-based advice to…

Evrysdi (risdiplam) was generally safe and well tolerated in a broader group of children and adults with spinal muscular atrophy (SMA) types 1 and 2 than that included in clinical trials supporting the therapy’s approval. These were the findings of an analysis of safety data from the U.S.