SMA Type 3: Symptoms, Treatment, and More - SMA News Today
Feb 2, 2023 · Type 3 is a milder form of spinal muscular atrophy (SMA), a rare genetic disease characterized by the progressive loss of motor neurons, the nerve cells that control voluntary movement. SMA results in muscle weakness and wasting among patients. One of five main forms of the disease, Type 3 is sometimes called juvenile SMA or Kugelberg-Welander syndrome.
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 ...
Spinal Muscular Atrophy (SMA) Treatment – SMA News Today
Feb 2, 2023 · There are several treatment options available for spinal muscular atrophy (SMA), a rare genetic condition characterized by progressive muscle weakness and wasting. The disease mainly affects motor function, but many patients also may experience breathing, swallowing, and speech difficulties, along with bone defects and other symptoms.
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.
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.
Zolgensma (onasemnogene abeparvovec-xioi) for SMA - SMA …
Apr 18, 2023 · A one-time gene therapy, Zolgensma (onasemnogene abeparvovec-xioi) is widely approved for the treatment of spinal muscular atrophy (SMA). It was the second SMA treatment ever to be approved in the U.S. and the first approved gene therapy for the rare disease. Zolgensma was originally developed by AveXis, now known as Novartis Gene Therapies ...
SMA Patients' Lives Getting Better with Kinova's Jaco Robot
Oct 20, 2017 · The robot reduces caregiver dependency by at least 41 percent, Clark said, adding that it’s used by a variety of patients, including those with spinal cord injuries, SMA, muscular dystrophy, ALS, and stroke. “A person with severe spastic cerebral palsy, for instance, puts out an incredible amount of effort to do the simplest task,” Clark ...
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 ...
Newborn Screening for SMA in 18 US States as Efforts Continue to...
Feb 12, 2020 · Eighteen states are now screening newborns for spinal muscular atrophy (SMA), the most recent neuromuscular disease added to a list of serious genetic disorders that infants can be tested for shortly after birth, advocacy officers at …
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 ...