Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial

Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

N Engl J Med.

Mercuri E, Darras BT, Chiriboga CA, Day JW, Campbell C, Connolly AM, Iannaccone ST, Kirschner J, Kuntz NL, Saito K, Shieh PB, Tulinius M, Mazzone ES, Montes J, Bishop KM, Yang Q, Foster R, Gheuens S, Bennett CF, Farwell W, Schneider E, De Vivo DC, Finkel RS

doi: 10.1056/NEJMoa1710504.

Aging and Cognitive Functions

Cognitive behavioural therapy with optional graded exercise therapy in patients with severe fatigue with myotonic dystrophy type 1: a multicentre, single-blind, randomised trial

Lancet Neurol.

Okkersen K, Jimenez-Moreno C, Wenninger S, Daidj F, Glennon J, Cumming S, Littleford R, Monckton DG, Lochmüller H, Catt M, Faber CG, Hapca A, Donnan PT, Gorman G, Bassez G, Schoser B, Knoop H, Treweek S, van Engelen BGM; OPTIMISTIC consortium

doi: 10.1016/

Migraine and Headache

A phase 3, long-term, open-label safety study of Galcanezumab in patients with migraine

BMC Neurol.

Camporeale A, Kudrow D, Sides R, Wang S, Van Dycke A, Selzler KJ, Stauffer VL

doi: 10.1186/s12883-018-1193-2.

Stroke

Intrathecal baclofen therapy versus conventional medical management for severe poststroke spasticity: results from a multicentre, randomised, controlled, open-label trial (SISTERS)

J Neurol Neurosurg Psychiatry.

Creamer M, Cloud G, Kossmehl P, Yochelson M, Francisco GE, Ward AB, Wissel J, Zampolini M, Abouihia A, Berthuy N, Calabrese A, Loven M, Saltuari L

doi: 10.1136/jnnp-2017-317021.

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Multiple Sclerosis

Safety and efficacy of tocilizumab versus azathioprine in highly relapsing neuromyelitis optica spectrum disorder (TANGO): an open-label, multicentre, randomised, phase 2 trial

★ 3 Recommended by Prof. Dr Christian Kamm, Faculty Member

doi: 10.1016/S1474-4422(20)30070-3.

Epilepsy

Safety and effectiveness of hormonal treatment versus hormonal treatment with vigabatrin for infantile spasms (ICISS): a randomised, multicentre, open-label trial

Lancet Neurol.

O'Callaghan FJ, Edwards SW, Alber FD, Hancock E, Johnson AL, Kennedy CR, Likeman M, Lux AL, Mackay M, Mallick AA, Newton RW, Nolan M, Pressler R, Rating D, Schmitt B, Verity CM, Osborne JP; participating investigators

doi: 10.1016/

Epilepsy

Final safety, tolerability, and seizure outcomes in patients with focal epilepsy treated with adjunctive perampanel for up to 4 years in an open-label extension of phase III randomized trials: Study 307

Epilepsia.

Krauss GL, Perucca E, Kwan P, Ben-Menachem E, Wang XF, Shih JJ, Patten A, Yang H, Williams B, Laurenza A

doi: 10.1111/epi.14044.

Anxiety and Depression

Esketamine Nasal Spray Plus Oral Antidepressant in Patients With Treatment-Resistant Depression: Assessment of Long-Term Safety in a Phase 3, Open-Label Study (SUSTAIN-2)

J Clin Psychiatry.

Wajs E, Aluisio L, Holder R, Daly EJ, Lane R, Lim P, George JE, Morrison RL, Sanacora G, Young AH, Kasper S, Sulaiman AH, Li CT, Paik JW, Manji H, Hough D, Grunfeld J, Jeon HJ, Wilkinson ST, Drevets WC, Singh JB

doi: 10.4088/JCP.19m12891.

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Migraine and Headache

Ubrogepant, an Acute Treatment for Migraine, Improved Patient-Reported Functional Disability and Satisfaction in 2 Single-Attack Phase 3 Randomized Trials, ACHIEVE I and II

★ 2 Recommended by Prof. Dr Christian Kamm, Faculty Member

★ 3 Recommended by Prof. Dr Claudio Gobbi, Faculty Member

Headache.

Dodick DW, Lipton RB, Ailani J, Halker Singh RB, Shewale AR, Zhao S, Trugman JM, Yu SY, Viswanathan HN

doi: 10.1111/head.13766.

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