Expertise in RNA Biology at Core of PTC’s Formative Work With Evrysdi
[Editor’s Note: This is one in a series of articles about the discovery and development of Evrysdi, the…
Articles by Marta Figueiredo, PhD
SMA Foundation’s ‘Little Toolbox’ Helped Pave Way to Therapies
[Editor’s Note: This is part of a series of articles into the discovery and development of Evrysdi, SMA’s…
‘Lucky Genetics’ and Hard Work: SMA’s Leap to 3 DMTs in 4 Years
[Editor’s Note: This is part of a series of articles into the discovery and development of Evrysdi, SMA’s…
FDA Approves Risdiplam, as Evrysdi, 1st Oral Treatment for All SMA Types
The U.S. Food and Drug Administration (FDA) has approved Evrysdi — formerly known as risdiplam — as the first…
New ASAH1 Mutations Linked to Rare Case of SMA and Farber Disease
A rare instance of a child with both spinal muscular atrophy (SMA) and Farber disease, associated with two new…
Spinraza-Zolgensma Combination Well-tolerated in Children with SMA Type 1, Study Shows
Combining Spinraza (nusinersen) with the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) is generally well-tolerated and sustains motor improvements in children…
Greatest Loss of Motor Skills at Ages 5 to 13 in SMA Type 2, Study Shows
People with type 2 spinal muscular atrophy (SMA) show the greatest loss of motor abilities on the Hammersmith Functional…
Biogen to Launch First Trial Testing Spinraza in Children Previously Given Zolgensma
Biogen has announced plans to launch a Phase 4 clinical trial evaluating the benefits of Spinraza (nusinersen) in…
‘Significant’ Limits to Treatment Seen for SMA Type 0 Baby in Case Report
A baby with spinal muscular atrophy (SMA) type 0 treated with both Spinraza (nusinersen) and Zolgensma is showing…
Motor Function Improved After 1 Year of Spinraza in Children With SMA Types 1–2
One year of Spinraza (nusinersen) treatment safely and significantly improves motor function in children with spinal muscular atrophy (SMA)…