Marta Figueiredo, PhD, science writer —

Marta holds a biology degree, a master’s in evolutionary and developmental biology, and a PhD in biomedical sciences from the University of Lisbon, Portugal. She was awarded a research scholarship and a PhD scholarship, and her research focused on the role of several signaling pathways in thymus and parathyroid glands embryonic development. She also previously worked as an assistant professor of an annual one-week embryology course at the University of Lisbon’s Faculty of Medicine.

Articles by Marta Figueiredo

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 to motor improvements in children with spinal muscular atrophy (SMA) type 1, according to a case series study. While children were followed for a short time after Evrysdi was…

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 evaluated in a Phase 1/2 clinical trial. According to the company’s announcement, this “difficult” decision was based on the “rapid advancements in the SMA treatment landscape in recent years,”…

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 adult-onset spinal muscular atrophy (SMA), according to a case report. While VRK1 mutations are mainly associated with pediatric-onset neuromuscular conditions marked by brain abnormalities and cognitive problems, these new cases…

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 with spinal muscular atrophy (SMA) type 3, according to real-world data from an international registry. Notably, when compared with an external group of untreated patients, who always showed negative changes in…

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 spinal muscular atrophy (SMA) type 1, according to data from a small Phase 1 clinical trial in Iran. Notably, the stem cell treatment appeared to have effectively slowed disease progression…

NICE Initially Against Adding Evrysdi to UK Public Health System

The initial position of England’s National Institute for Health and Care Excellence (NICE) is to recommend against adding Roche’s Evrysdi (risdiplam) to the list of medications available to spinal muscular atrophy (SMA) patients through the country’s national health service (NHS). Lack of evidence of Evrysdi’s efficacy in babies genetically diagnosed…