2 SMN2-targeting Therapies Work Better Than 1 in Mouse Model
Treatment with two distinct molecules targeting the “backup” SMN2 gene resulted in higher levels of SMN — the protein lacking…
Articles by Marta Figueiredo, PhD
Most Parents Surveyed in Japan Support SMA Newborn Screening
Despite being mostly unaware of spinal muscular atrophy (SMA), most Japanese parents support newborn screening for the disease, according…
SMA Type 1 Affects Sensory Nerves as Children Age, Small Study Shows
Sensory nerves — those responsible for transmitting sensory information — are progressively damaged in children with spinal muscular atrophy…
‘Best Possible Outcome’ for SMA? Newborn Screening, Then Zolgensma
Despite its high cost, giving the one-time gene therapy Zolgensma to an infant identified through newborn screening with spinal…
Zolgensma-Evrysdi Combo Likely Beneficial for SMA Type 1
Daily treatment with Evrysdi (risdiplam) following the one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) is generally well-tolerated and leads…
Novartis Stopping Work on Branaplam as Oral SMA Therapy
Novartis is stopping the clinical development of branaplam (LMI070), its experimental oral treatment for spinal muscular atrophy (SMA) being…
Adult-onset SMA Form, With VRK1 Mutations, Found in 2 Hispanics
Two mutations in the VRK1 gene were found in two unrelated Hispanic adults, with symptoms suggestive of an atypical form of…
Spinraza Improves Motor Function in SMA Type 3 Children, Too
About one year of treatment with Spinraza (nusinersen) effectively prevents or reverses motor function decline in children and adults…
Stem Cell Therapy May Benefit Some Type 1 Infants
Treatment with human fat tissue-derived mesenchymal stem cells (MSCs) may improve motor nerve cell health and survival in infants with…
Newborn Screening in Massachusetts Seen as Highly Accurate, Effective
Newborn screening (NBS) for spinal muscular atrophy (SMA) in Massachusetts allowed for early detection and treatment of affected infants, with…