Basil T. Darras

发表论文 524 篇 · 总被引 23384 次 · h-index 74

代表论文

Determining minimal clinically important differences in the Hammersmith Functional Motor Scale Expanded for untreated spinal muscular atrophy patients: An international study (2024 · European Journal of Neurology · 被引 34)
Spinal Muscular Atrophy Update in Best Practices (2024 · Neurology Clinical Practice · 被引 33)
Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trial (2025 · The Lancet Neurology · 被引 30)
Hepatocyte-intrinsic SMN deficiency drives metabolic dysfunction and liver steatosis in spinal muscular atrophy (2024 · Journal of Clinical Investigation · 被引 29)
Safety and Efficacy of IV Onasemnogene Abeparvovec for Pediatric Patients With Spinal Muscular Atrophy (2024 · Neurology · 被引 28)
Long-term efficacy, safety, and patient-reported outcomes of apitegromab in patients with spinal muscular atrophy: results from the 36-month TOPAZ study (2024 · Frontiers in Neurology · 被引 20)