Advocates come together for SMA Awareness Month in August
Jul 31, 2024 · August is Spinal Muscular Atrophy (SMA) Awareness Month, and patients, caregivers, doctors, researchers, and other supporters are coming together to improve …
HCP SMA News Today – The Web's Leading Resource For People …
Oct 10, 2018 · SBMA therapy NIDO-361 granted orphan drug status in Europe. NIDO-361, Nido Biosciences’ experimental oral treatment for spinal and bulbar muscular atrophy (SBMA) — a …
SMA treatment Zolgensma seen to benefit patients in real world
Sep 13, 2024 · Treatment with Zolgensma (onasemnogene abeparvovec-xioi) improves motor function in children with spinal muscular atrophy (SMA) who carry at least four copies of the so …
Family History Can Be Key in Identifying Silent Carriers of SMA
Jan 7, 2022 · Notably, the number of copies of a “backup” SMN gene, SMN2, influences SMA severity, with a higher number typically predicting milder disease. While people with only one …
Long-term Spinraza found safe, effective across SMA spectrum
Feb 14, 2024 · Long-term treatment with Spinraza (nusinersen) was associated with improved or stable motor function in patients across the spinal muscular atrophy (SMA) disease spectrum, …
Spinal Muscular Atrophy: Cardiac Disease – HCP SMA News Today
Spinal Muscular Atrophy (SMA) is an inherited neurodegenerative disease, 1 but it is increasingly clear that the pathology of SMA extends beyond ventral horn spinal cord neurons and includes …
Real-world benefits of Zolgensma for SMA seen in RESTORE data
Jan 31, 2024 · RESTORE (NCT04174157) is a real-world, observational patient registry that’s tracking outcomes for up to 15 years and seeks to capture responses to DMTs that weren’t …
Spinraza, in Range of SMA Patients, Shows Efficacy Over Years of Use
May 20, 2020 · “As the first approved treatment for SMA, Spinraza offers a significant data set that allows us to uniquely assess the safety and durability of repeated doses over time …
Types of spinal muscular atrophy - SMA News Today
Feb 2, 2023 · Spinal muscular atrophy, or SMA, is a genetic disorder that is divided into five main types based on the age at which symptoms first appear. These forms of SMA range from type …
Zolgensma, 1st Gene Therapy to Treat SMA, Approved for …
May 24, 2019 · The FDA decision was based on data from the ongoing Phase 3 STR1VE trial (NCT03306277), and the completed Phase 1 START trial (NCT02122952) evaluating the …