MicroRNAs in Blood May Predict Spinzara Response in Type 1 Patients
MicroRNAs isolated from the bloodstream of patients with infant-onset spinal muscular atrophy (SMA) type 1 before treatment were related to functional responses to Spinraza (nusinersen) after six months of therapy, a study suggested. Researchers said these microRNAs...
Neurologists Confident SMA Treatments Help Youngest Patients Most
Pediatric neurologists’ expectations in terms of treatment benefits are decidedly higher for infants and toddlers with spinal muscular atrophy (SMA) types 1 and 2 than for adolescent patients, a study in Spain shows. Notably, neurologists with low aversion to...
Medical Costs Lower if Treatment Is Started Before Symptoms: Study
The total medical costs associated with spinal muscular atrophy (SMA) are lower for patients diagnosed at birth via newborn screening, compared with those diagnosed after they start showing symptoms of the disease, a new study has found. These findings “demonstrate...
Manitoba Newborn Screening Program Expands to Include SMA
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...
Higher Spinraza Dose May Be More Effective, Modeling Study Predicts
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...HCP — SMA News Today
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