José Lopes, PhD,  managing science editor—

Babies with spinal muscular atrophy (SMA) treated with Zolgensma before experiencing symptoms continue to reach motor milestones in a normal timeframe, according to updated Phase 3 results. “With genetic testing in hand and presymptomatic treatment, we can look forward to a future in which age-appropriate motor milestone development…

A new global Phase 2/3 clinical trial will test whether higher doses of Spinraza (nusinersen) provide greater efficacy to spinal muscular atrophy (SMA) patients of all ages. Biogen aims to include 125 participants with infantile- or later-onset SMA in the DEVOTE study (NCT04089566), to be…

With increasing insurance coverage and AveXis’ plans to broaden access to older spinal muscular atrophy (SMA) patients, the U.S. Food and Drug Administration (FDA) remains “very confident” in the use of Zolgensma despite preclinical data concerns, according to an official from the agency. Cure SMA…

A new and faster genetic test for spinal muscular atrophy (SMA), called SMA STAT, will be offered free of charge in the U.S. as part of the SMA Identified program. According to Biogen and Invitae Corporation, SMA STAT reduces the time for a definitive SMA diagnosis — through…

Skye, a nine-month-old baby with spinal muscular atrophy (SMA) type 1, is reaching new milestones every week and is “the happiest child” after being treated with Zolgensma, according to her mother Kasia. “She is almost sitting, her head and neck are really strong, and she is reaching above…

The European Medicines Agency (EMA) has granted orphan medicinal product designation to reldesemtiv, a potential add-on therapy for patients with spinal muscular atrophy (SMA). Reldesemtiv, developed by Cytokinetics and Astellas, is meant to complement existing SMA treatments. It is a fast skeletal muscle troponin activator…

A new long-term partnership with Catalent Biologics will allow Zolgensma’s developer AveXis to increase production of the gene therapy and gain access to further technical expertise. Zolgensma was approved by the U.S. Food and Drug Administration (FDA) to treat pre-symptomatic newborns through 2-year-olds with any type…

Treating teenagers and adults with oral risdiplam, a potential therapy for spinal muscular atrophy (SMA), led to sustained increases in blood levels of the key SMN protein missing in these patients, the lead investigator for a Phase 2 clinical trial said in an interview. Claudia A. Chiriboga, MD,…

The European label for Spinraza (nusinersen) has been updated to include longer-term benefits seen in pre-symptomatic treatment of spinal muscular atrophy (SMA), as well as in patients with infantile- and later-onset forms of the disease. Overall, the results from four clinical trials show sustained lessening or…

Presymptomatic infants treated with Spinraza (nusinersen) are able to hit motor milestones that are otherwise unachievable in the natural progression of their condition, spinal muscular atrophy (SMA), with all treated children able to sit without support and most to walk unassisted in a normal timeframe, according to…