Long-Term Effects of Antidarsagene Autotemcel for Metachromatic Leukodystrophy
NEJM 392:1609-1620, Fumagalli,F.,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
Safety and Tolerability of Gene Therapy With an Adeno-Associated Virus (AAV) Borne GAD gene for Parkinsons Disease: An Open Label, Phase I Trial
Lancet 369:2097-2105,2056, Kaplitt,M.G.,et al, 2007
Calcitonin Gene-Related Peptide-Targeted Therapy in Migraine: Current Role and Future Perspectives
Lancet 405:1014-1026, Versijpt,J.,et al, 2025
Intravenous Thrombolysis Use Before Inter-Facility Transfer for Thrombectomy:Association with Efficacy and Safety Outcomes
Ann Neurol 98:871-880, Seners,P.,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
Pomalidomide for Epistaxis in Hereditary Hemorrhagic Telangiectasia
NEJM 391:1015-1027, Al-Samkari,H.,et al, 2024
Atogepant for the Preventive Treatment of Chronic Migraine (PROGRESS): A Randomised, Double-blind, Placebo-controlled, Phase 3 Trial
Lancet 402:775-785, Pozo-Rosich,P.,et al, 2023
Acute Treatment for Migraine
JAMA 325:2346-2347, Burch, R. & Rayhill, M., 2021
Atogepant for the Preventive Treatment of Migraine
NEJM 385:695-706, Ailani,J.,et al, 2021
Remigepant, an Oral Calcitonin Gene-Related Peptide Receptor Antagonist, for Migraine
NEJM 381:142-149, Lipton, R.B.,et al, 2019
Trial of Galcanezumab in Prevention of Episodic Cluster Headache
NEJM 381:132-141, Goadsby, P.J.,et al, 2019
Targeting Calcitonin Gene-Related Peptide: A New Era in Migraine Therapy
Lancet 394:1765-1774, Charles, A. & Pozo-Rosich, P., 2019
Ubrogepant for the Treatment of Migraine
NEJM 381:2230-2241, Dodick, D.W.,et al, 2019
Efficacy and Tolerability of Erenumab in Patients with Episodic Migraine in Whom Two-of-Four Previous Preventive Treatments were Unsuccessful: A Randomised, Double-Blind, Placebo-Controlled, Phase 3b Study
Lancet 392:2280-2287,2241, Reuter, U.,et al, 2018
Galcanezumab in Chronic Migraine
Neurol 91:e2211-e2221, Detke, H.C.,et al, 2018
CGRP - The Next Frontier for Migraine
NEJM 377:2190-2191, Hershey, A.D., 2017
Efficacy and Safety of Everolimus for Subependymal Giant Cell Astrocytomas Associated with Tuberous Sclerosis Complex (EXIST-1): A Multicenter, Randomized, Placebo-Controlled Phase 3 Trial
Lancet 381:125-132, Franz, D.,et al, 2013
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