February 01, 2026

#spinal muscular atrophy — Sunday, February 01, 2026 (2 items)

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0		💬 0	Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial. (nature.com)	Kwon JM … van der Pol WL Nature Medicine 2026-02-01	#gene therapy #spinal muscular atrophy #safety #clinical trial
0		💬 0	Intrathecal onasemnogene abeparvovec in treatment-naive patients with spinal muscular atrophy: a phase 3, randomized controlled trial. (nature.com)	Proud CM … Finkel RS Nature Medicine 2026-02-01	#gene therapy #spinal muscular atrophy #clinical trial #motor function