Long-Term Effects of Antidarsagene Autotemcel for Metachromatic Leukodystrophy
NEJM 392:1609-1620, Fumagalli,F.,et al, 2025
Effectiveness and Safety of IV Thrombolysis Before Hospital Transfer for Thrombectomy in Patients With Basilar Artery Occlusion
Neurol 105:e214355, Liebart,S.,et al, 2025
Lentiviral Haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access
Lancet 399:372-383, Fumagalli, F.,et al, 2022
Hematopoietic Stem - and Progenitor-Cell Gene Therapy for Hurler Syndrome
NEJM 385:1929-1940, Gentner, B.,et al, 2021
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
NEJM 377:1713-1722,1786, Mendell, J.R.,et al, 2017
Long-Term Safety and Tolerability of ProSavin, a Lentiviral Vector-Based Gene Therapy for Parkinsons Disease: A Dose Escalation, Open-Label, Phase 1/2 Trial
Lancet 383:1138-1146,1107, Palfi, S.,et al, 2014
Does Helicopter Emergency Medical Service Transfer Offer Benefit to Patients with Stroke?
Stroke 43:878-880, Olson,M.D. and Rabinstein,A.A., 2012
Guidelines for the Early Management of Adults With Ischemic Stroke
Stroke 38:1655-1711, Adams,H.P.,et al, 2007