Mar 27, 2025 · Novartis now developing OAV101 gene therapy for older SMA patients. Data from clinical trials and real-world studies have consistently shown that Zolgensma and other …

5 days ago · SMA gene therapy designed to aid motor neurons. GC101 uses a harmless adeno-associated virus serotype 9 (AAV9) to carry a healthy copy of the SMN1 gene that has been …

Mar 28, 2025 · MDA 2025: SMA gene therapy ‘transformational,’ per Novartis exec. It’s been almost six years since the one-time gene therapy Zolgensma (onasemnogene abeparvovec …

Apr 16, 2025 · Building an SMA support systemcan make all the difference. Whether you need emotional support, practical advice, or the comfort of knowing someone else gets it, …

Mar 19, 2025 · The first clinical trial testing Zolgensma, launched in 2014, was called START (NCT02122952).It tested the intravenous, or into-the-vein, therapy in 15 babies with SMA type …

Spinal Muscular Atrophy (SMA) is a congenital neuromuscular disorder characterized most prominently by progressive muscle atrophy and weakness. 1 SMA is a neurodegenrative …

Mar 24, 2025 · Results from the study, which was sponsored by Spinraza’s maker Biogen, were presented at the annual meeting of the Muscular Dystrophy Association (MDA) in the poster, “ …

May 14, 2025 · SMA gene therapy GC101 found safe, effective in animals. Gene therapy GC101 was found to rescue disease manifestations in a mouse model of spinal muscular atrophy …

May 12, 2025 · By the grace of God, takeout, and copious amounts of television, we made it through. Treatment went better than anticipated. We celebrated Christmas with less fanfare …

DMTs boost fine motor skills in young SMA patients able to sit. A year of disease-modifying therapies (DMTs) improved motor function, particularly fine motor skills, in people with spinal …