Search details
1.
Communicating hydrocephalus and raised intracranial pressure in association with multi-systemic smooth muscle dysfunction syndrome (MSMDS).
Childs Nerv Syst
; 39(3): 825-828, 2023 03.
Article
in English
| MEDLINE | ID: mdl-36271050
2.
Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.
JAMA
; 327(15): 1456-1468, 2022 04 19.
Article
in English
| MEDLINE | ID: mdl-35381069
3.
Pathogenic variants in MT-ATP6: A United Kingdom-based mitochondrial disease cohort study.
Ann Neurol
; 86(2): 310-315, 2019 08.
Article
in English
| MEDLINE | ID: mdl-31187502
4.
Utility and safety of plasma exchange in paediatric neuroimmune disorders.
Dev Med Child Neurol
; 61(5): 540-546, 2019 05.
Article
in English
| MEDLINE | ID: mdl-30659589
5.
Development of a consensus core dataset in juvenile dermatomyositis for clinical use to inform research.
Ann Rheum Dis
; 77(2): 241-250, 2018 02.
Article
in English
| MEDLINE | ID: mdl-29084729
6.
British Society for Rheumatology guideline on management of paediatric, adolescent and adult patients with idiopathic inflammatory myopathy.
Rheumatology (Oxford)
; 61(5): 1760-1768, 2022 05 05.
Article
in English
| MEDLINE | ID: mdl-35355064
7.
Migrating partial seizures of infancy: expansion of the electroclinical, radiological and pathological disease spectrum.
Brain
; 136(Pt 5): 1578-91, 2013 May.
Article
in English
| MEDLINE | ID: mdl-23599387
8.
Risdiplam in Spinal Muscular Atrophy: Safety Profile and Use Through The Early Access to Medicine Scheme for the Paediatric Cohort in Great Britain.
J Neuromuscul Dis
; 11(2): 361-368, 2024.
Article
in English
| MEDLINE | ID: mdl-38189761
9.
Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
Lancet Neurol
; 23(4): 393-403, 2024 Apr.
Article
in English
| MEDLINE | ID: mdl-38508835
10.
Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy: A Randomized Controlled Trial.
Neurology
; 102(5): e208112, 2024 Mar 12.
Article
in English
| MEDLINE | ID: mdl-38335499
11.
An investigation into the relationship between vigabatrin, movement disorders, and brain magnetic resonance imaging abnormalities in children with infantile spasms.
Dev Med Child Neurol
; 55(9): 862-7, 2013 Sep.
Article
in English
| MEDLINE | ID: mdl-23789722
12.
Safety and efficacy of tamoxifen in boys with Duchenne muscular dystrophy (TAMDMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
Lancet Neurol
; 22(10): 890-899, 2023 10.
Article
in English
| MEDLINE | ID: mdl-37739572
13.
Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.
JAMA Neurol
; 79(10): 1005-1014, 2022 10 01.
Article
in English
| MEDLINE | ID: mdl-36036925
14.
Preventing Cardiomyopathy in DMD: A Randomized Placebo-Controlled Drug Trial.
Neurol Clin Pract
; 11(5): e661-e668, 2021 Oct.
Article
in English
| MEDLINE | ID: mdl-34840880
15.
A Phase 1b Trial to Assess the Pharmacokinetics of Ezutromid in Pediatric Duchenne Muscular Dystrophy Patients on a Balanced Diet.
Clin Pharmacol Drug Dev
; 8(7): 922-933, 2019 10.
Article
in English
| MEDLINE | ID: mdl-30650257
16.
Randomised placebo-controlled trial of combination ACE inhibitor and beta-blocker therapy to prevent cardiomyopathy in children with Duchenne muscular dystrophy? (DMD Heart Protection Study): a protocol study.
BMJ Open
; 8(12): e022572, 2018 12 19.
Article
in English
| MEDLINE | ID: mdl-30573480
17.
A checklist for clinical trials in rare disease: obstacles and anticipatory actions-lessons learned from the FOR-DMD trial.
Trials
; 19(1): 291, 2018 May 10.
Article
in English
| MEDLINE | ID: mdl-29793540
18.
Developing standardized corticosteroid treatment for Duchenne muscular dystrophy.
Contemp Clin Trials
; 58: 34-39, 2017 07.
Article
in English
| MEDLINE | ID: mdl-28450193
19.
Pain experience, expression and coping in boys and young men with Duchenne Muscular Dystrophy - A pilot study using mixed methods.
Eur J Paediatr Neurol
; 20(4): 630-8, 2016 Jul.
Article
in English
| MEDLINE | ID: mdl-27053141
20.
Safety, Tolerability, and Pharmacokinetics of SMT C1100, a 2-Arylbenzoxazole Utrophin Modulator, following Single- and Multiple-Dose Administration to Pediatric Patients with Duchenne Muscular Dystrophy.
PLoS One
; 11(4): e0152840, 2016.
Article
in English
| MEDLINE | ID: mdl-27055247