Spinal Muscular Atrophy: Nutrition and Supplementation
WEBThe nutritional management of patients with progressive neuromuscular diseases like Spinal Muscular Atrophy (SMA) is complex and is achieved optimally with an individualized approach from a multidisciplinary care team. 1,2 Patients with SMA need nutritional monitoring at least annually with assessment of caloric intake and standard ...
Advocates come together for SMA Awareness Month in August
WEBJul 31, 2024 · August is Spinal Muscular Atrophy (SMA) Awareness Month, and patients, caregivers, doctors, researchers, and other supporters are coming together to improve disease knowledge, patient advocacy, and research funding to help find a cure for the rare genetic condition. SMA is a neuromuscular disorder that …
HCP SMA News Today – The Web's Leading Resource For People …
WEBTreatment with Spinraza (nusinersen) generally stabilized or improved motor function for older patients with spinal muscular atrophy (SMA), according to a review and meta-analysis of previously published observational studies. The new analysis, which spanned a treatment period of up to two years, involved adults and adolescents with….
SMA Type 4: Symptoms, Treatment, and More - SMA News Today
WEBFeb 2, 2023 · 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. Prognosis. Lifespan is unaffected, and most patients retain the ability to walk independently.
Next-generation SMA gene therapy is goal of Voyager, Novartis
WEBby Andrea Lobo January 8, 2024. Voyager Therapeutics is collaborating with Novartis to discover and develop next-generation gene therapies for conditions like spinal muscular atrophy (SMA). Under terms of the agreement, Novartis will obtain a target-exclusive license to Voyager’s TRACER capsids for SMA and Huntington’s disease, and it will ...
FAQs About Evrysdi (Risdiplam) - SMA News Today
WEBAug 7, 2020 · FAQs About Evrysdi (Risdiplam) by Grace Frank August 7, 2020. Evrysdi (risdiplam) is an oral liquid treatment approved in the U.S. for infants through adults with spinal muscular atrophy (SMA) — patients age 2 months and older with any disease type. Roche and Genentech developed Evrysdi in collaboration with …
SMA Type 2: Symptoms, Treatment, and More - SMA News Today
WEBFeb 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 …
With Zolgensma Approval for SMA, Debate Shifts to Pricing...
WEBMay 29, 2019 · Zolgensma, approved for children with spinal muscular atrophy (SMA) up to 2 years old, has two claims to fame: It is both the first gene therapy ever developed to treat a neuromuscular disease, and it’s by far the world’s most expensive drug. At $2.125 million, a 60-minute intravenous infusion of Zolgensma costs more than a 2,000-square ...
Spinal Muscular Atrophy: Caregiver Support – HCP SMA News Today
WEBCaregivers of patients with Spinal Muscular Atrophy (SMA) report neglected aspects of their lives. 1 Caregivers will need different kinds of support depending on factors such as the specific type of SMA their child has, whether they have other children, whether they intend to have future pregnancies, their socioeconomic status, and how ...
SMA Type 3: Symptoms, Treatment, and More - SMA News Today
WEBFeb 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.