No new safety signals related to Evrysdi (risdiplam) were documented in a compassionate use program in Germany where more than 100 people with spinal muscular atrophy (SMA) were treated with the oral therapy prior to its approval last year, a new study reports. “We present the first real-world…

Spinraza (nusinersen) treatment was generally well tolerated and led to improved or stabilized motor function for nearly 300 children with spinal muscular atrophy (SMA) in Poland, a new study reports. The study, “Safety, tolerability, and efficacy of a widely available nusinersen program for Polish children with Spinal Muscular…

A Phase 3 clinical trial evaluating Biohaven Pharmaceuticals’ taldefgrobep alfa as an add-on therapy in children and young adults with spinal muscular atrophy (SMA) has enrolled its first patient. The trial, dubbed RESILIENT (NCT05337553), expects to recruit up to 180 SMA patients already on disease-modifying therapies (DMTs), and…

A single lyric keeps ringing in my head from Fletcher’s latest single, “Her Body Is Bible.” It goes, “Hold on tight, when the world gets hard, this s**t’s like paradise.” The song appears pretty straightforward at first blush. The lyrics and the imagery are simple, attempting to reclaim religious…

Two years of treatment with the investigational therapy apitegromab continued to improve motor function in Spinraza-treated children and young adults with later-onset spinal muscular atrophy (SMA) who cannot walk, according to new extension data of the Phase 2 TOPAZ trial. The therapy’s developer, Scholar Rock, is also…

The Australian state of Queensland is expanding its newborn screening program to include the genetic conditions spinal muscular atrophy (SMA) and severe combined immunodeficiency (SCID). New testing capabilities are expected to be in place by next May, with the free screening to begin soon after. Queensland, Australia’s second-largest state,…

The video opens with Blake Shofner driving and saying to his younger brother, Nolan, “Dang Nolan, these roads are terrible.” Nolan Shofner, who has spinal muscular atrophy (SMA) type 1, replies in a southern accent with a computerized voice: “My bones are bouncing all over the place.” With…

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 clearly that as long as the decision to reimburse…

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…

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…