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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.
Zolgensma appears generally safe and well tolerated among spinal muscular atrophy (SMA) patients weighing 8.5 kilograms (about 18.7 lbs) or more, with no new safety concerns identified in the heavier group. Notably, 8.5 kilograms was the maximum weight of SMA patients enrolled in clinical trials supporting Zolgensma’s approval.
From being a child actor to a lawyer in the entertainment industry, Alexa Dectis, who has relied on a power wheelchair to get around since she was 2, has gone far. Dectis, diagnosed with spinal muscular atrophy (SMA) type 2 when she was 18 months old, was named…
Treatment with Spinraza (nusinersen) modestly improved muscle strength and stabilized motor function for adults with spinal muscular atrophy (SMA) types 2 or 3, according to a study from Israel. The study, “Longer-term follow-up of nusinersen efficacy and safety in adult patients with spinal muscular atrophy…
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