Detalhe da pesquisa
1.
Amifampridine phosphate (Firdapse(®)) is effective and safe in a phase 3 clinical trial in LEMS.
Muscle Nerve
; 53(5): 717-25, 2016 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-26852139
2.
Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02).
J Neurol
; 271(4): 1787-1801, 2024 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-38057636
3.
Long-term safety and efficacy of zilucoplan in patients with generalized myasthenia gravis: interim analysis of the RAISE-XT open-label extension study.
Ther Adv Neurol Disord
; 17: 17562864241243186, 2024.
Artigo
em Inglês
| MEDLINE | ID: mdl-38638673
4.
Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study.
Lancet Neurol
; 22(5): 395-406, 2023 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-37059508
5.
Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophy.
Ann Neurol
; 67(6): 771-80, 2010 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-20517938
6.
Efficacy and Safety of Bimagrumab in Sporadic Inclusion Body Myositis: Long-term Extension of RESILIENT.
Neurology
; 96(12): e1595-e1607, 2021 03 23.
Artigo
em Inglês
| MEDLINE | ID: mdl-33597289
7.
Long-term safety and tolerability of bimagrumab (BYM338) in sporadic inclusion body myositis.
Neurology
; 95(14): e1971-e1978, 2020 10 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-32690797
8.
Congenital myasthenic syndrome caused by a frameshift insertion mutation in GFPT1.
Neurol Genet
; 6(4): e468, 2020 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-32754643
9.
Safety and efficacy of intravenous bimagrumab in inclusion body myositis (RESILIENT): a randomised, double-blind, placebo-controlled phase 2b trial.
Lancet Neurol
; 18(9): 834-844, 2019 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-31397289
10.
Phenotypic spectrum of disorders associated with glycyl-tRNA synthetase mutations.
Brain
; 128(Pt 10): 2304-14, 2005 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-16014653
11.
Childhood-onset spastic paraplegia with NIPAL gene mutation.
J Child Neurol
; 21(11): 974-7, 2006 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-17092466
12.
Mortality and Causes of Death in Patients with Sporadic Inclusion Body Myositis: Survey Study Based on the Clinical Experience of Specialists in Australia, Europe and the USA.
J Neuromuscul Dis
; 3(1): 67-75, 2016 03 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-27854208
13.
Progressive skeletal myopathy, a phenotypic variant of desmin myopathy associated with desmin mutations.
Neuromuscul Disord
; 13(3): 252-8, 2003 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-12609507
14.
Charcot-Marie-Tooth disease: extensive cranial nerve involvement on CT and MR imaging.
AJNR Am J Neuroradiol
; 25(3): 494-7, 2004 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-15037479
15.
Treatment of sporadic inclusion body myositis with bimagrumab.
Neurology
; 83(24): 2239-46, 2014 Dec 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-25381300
16.
Lower extremity predominant stiff-person syndrome and limbic encephalitis with amphiphysin antibodies in breast cancer.
J Clin Neuromuscul Dis
; 14(2): 72-4, 2012 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-23172386
17.
Autosomal recessive spastic ataxia of Charlevoix-Saguenay: compound heterozygotes for nonsense mutations of the SACS gene.
J Child Neurol
; 26(12): 1585-9, 2011 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-21745802
18.
Painful brachial plexopathies in SEPT9 mutations: adverse outcome related to comorbid states.
J Clin Neuromuscul Dis
; 9(4): 379-84, 2008 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-18525421
19.
Histopathologic progression and a novel mutation in a child with nemaline myopathy.
J Child Neurol
; 23(7): 813-7, 2008 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-18487519
20.
Arterial thrombosis induced by IVIg and its treatment with tPA.
Neurology
; 60(11): 1825-6, 2003 Jun 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-12796540