Apitegromab (SRK-015) for spinal muscular atrophy - SMA News …
Jul 18, 2024 · Apitegromab (SRK-015) is an experimental muscle-directed infusion therapy being developed by Scholar Rock as an add-on treatment to improve motor function in people with spinal muscular atrophy (SMA). Apitegromab has received fast track status and orphan drug and rare pediatric disease designations by the U.S. Food and Drug Administration (FDA).
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 available in earlier registries. Here, the scientists described clinical outcomes for 168 registry participants who’d been treated with Zolgensma as of May 2022 and hadn’t received any prior SMA therapies.
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 the heart among other organs. 2 In patients with SMA, tissue levels of SMN1 protein are reduced in heart tissues, 3 and numerous mouse models of SMA exhibit cardiac developmental abnormalities which ...
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 disease type marked by progressive weakness in the muscles of the throat and mouth, and those closer to the trunk —…. October 21, 2024 News by Lindsey Shapiro PhD.
Specialized equipment for spinal muscular atrophy - SMA News …
Feb 6, 2023 · Wheelchairs are mobile chairs, either manual or powered, for people with spinal muscular atrophy (SMA). Wheelchairs can be pushed by a caregiver or rolled by the person with SMA. Power chairs can be controlled with a joystick, a head array or chin control, and offer options such as postural realignment, and pressure relief.
Oral SBMA therapy shows safety and tolerability in Phase 1 trial
Nov 15, 2023 · NIDO-361, Nido Biosciences ’ experimental oral treatment for spinal and bulbar muscular atrophy (SBMA), was seen to be generally safe and well tolerated in healthy adults who participated in an initial clinical trial. Results of the Phase 1 study (ACTRN12622000964718) were presented during the 2023 International Kennedy’s Disease Patient ...
SMA Type 2: Symptoms, Treatment, and More - SMA News Today
Feb 2, 2023 · Muscle weakness also can affect muscles in the chest, throat, and mouth, giving rise to other SMA type 2 symptoms that affect daily living. Such symptoms include: problems eating, due to weakness in the muscles used to chew and swallow. breathing and coughing difficulties resulting from chest muscle weakness, which can increase the risk of lung ...
SMA Type 4: Symptoms, Treatment, and More - SMA News Today
Feb 2, 2023 · Unlike the other four main types of SMA, type 4 rarely affects patients’ swallowing and breathing functions. SMA type. Type 4. Age of onset. During adulthood, usually after age 35. Symptoms. Progressive muscle weakness, usually starting in the legs. Fatigue, numbness, and cramping. Mild hand and finger tremors.
SMA News Today Home | SMA News Today
Nov 6, 2024 · Oral strength and endurance — important for functions like swallowing, speaking, and chewing — were found diminished in adults with spinal muscular atrophy (SMA) in three out of five assessments, according to a new study. In particular, untreated SMA type 2 patients who couldn’t walk had the most…. October 30, 2024 News by Marisa Wexler ...
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, according to an analysis of real-world registry data. “To date, this is the largest prospective study over the longest observational period of [Spinraza] therapy in adult ...