Therapy update in nerve, neuromuscular junction and myopathic disorders

Current Opinion in Neurology

Volumn 23, Issue 5, 2010, Pages 496-501

  Sadeghian, Hamid ^a   Wolfe, Gil I ^a  

^a UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER   (United States)

Author keywords

immunotherapy; myopathies; neuromuscular disorders; neuromuscular junction

Indexed keywords

3,4 DIAMINOPYRIDINE; ALEMTUZUMAB; ALPHA SARCOGLYCAN; ALPHA TOCOPHEROL; ANTISENSE OLIGODEOXYNUCLEOTIDE; ASCORBIC ACID; AZATHIOPRINE; CORTICOSTEROID; DEXAMETHASONE; EPHEDRINE; FLUOXETINE; IMMUNOGLOBULIN; METHOTREXATE; METHYLPREDNISOLONE; MONARSEN; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; PREDNISOLONE; QUINIDINE; RITUXIMAB; TACROLIMUS; UNCLASSIFIED DRUG; VINCRISTINE;

CHEMOTHERAPY INDUCED NEUROPATHY; CHEMOTHERAPY INDUCED POLYNEUROPATHY; CHRONIC INFLAMMATORY DEMYELINATING POLYNEUROPATHY; CONGENITAL MYASTHENIC SYNDROME; EXON SKIPPING; GENE THERAPY; GENE TRANSFER; HEREDITARY MOTOR SENSORY NEUROPATHY; HUMAN; IMMUNOTHERAPY; LAMBERT EATON MYASTHENIC SYNDROME; MULTIFOCAL MOTOR NEUROPATHY; MUSCULAR DYSTROPHY; MYASTHENIA GRAVIS; MYOPATHY; MYOSITIS; NEUROMUSCULAR JUNCTION DISORDER; NEUROPATHY; NEUROPROTECTION; NONHUMAN; POLYNEUROPATHY; REVIEW;

HUMANS; MUSCULAR DISEASES; NEUROMUSCULAR DISEASES; NEUROMUSCULAR JUNCTION DISEASES;

EID: 77956392274     PISSN: 13507540     EISSN: None     Source Type: Journal    
DOI: 10.1097/WCO.0b013e32833d7367     Document Type: Review

References (38)

1. Pareyson D, Marchesi C. Diagnosis, natural history, and management of Charcot-Marie-Tooth disease. Lancet Neurol 2009; 8:654-667.
2. Passage E, Norreel JC, Noack-Fraissignes P, et al. Ascorbic acid treatment corrects the phenotype of a mouse model of Charcot-Marie-Tooth disease. Nat Med 2004; 10:396-401.
3. Burns J, Ouvrier RA, Yiu EM, et al. Ascorbic acid for Charcot-Marie-Tooth disease type 1A in children: a randomized, double-blind, placebo-controlled, safety and efficacy trial. Lancet Neurol 2009; 8:537-544.
4. Micallef J, Attarian S, Dubourg O, et al. Effect of ascorbic acid in patients with Charcot-Marie-Tooth type 1A: a multicenter, randomized, double-blind, placebo controlled trial. Lancet Neurol 2009; 8:1103-1110.
5. Hughes RAC, Bouche P, Cornblath DR, et al. EFNS/PNS guideline on management of chronic inflammatory polyradiculoneuropathy: report of a joint task force of the European Federation of Neurological Societies and the Peripheral Nerve Society. Eur J Neurol 2006; 13:326-332.
6. van Schaik IN, Eftimov P, van Doorn PA, et al. Pulsed high-dose dexamethasone versus standard prednisolone treatment for chronic inflammatory demyelinating polyradiculoneuropathy (PREDICT study): a double-blind, randomized, controlled trial. Lancet Neurol 2010; 9:245-253.
7. Bedi G, Brown A, Tong T, Sharma KR. Chronic inflammatory demyelinating polyneuropathy responsive to mycophenolate mofetil therapy. J Neurol Neurosurg Psychiatry 2010; 81:634-636.
8. Meuth SG, Kleinschnitz C. Multifocal motor neuropathy: update on clinical characteristics, pathophysiological concepts and therapeutic options. Eur Neurol 2010; 63:193-204.
9. Baumann A, Hess CW, Sturzenegger M. IVIg dose increase in multifocal motor neuropathy: a prospective six month follow-up. J Neurol 2009; 256:608-614.
10. Pace A, Giannarelli D, Galie; E, et al. Vitamin E neuroprotection for cisplatin neuropathy: a randomized, placebo-controlled trial. Neurology 2010; 74:762-766.
11. Skeie GO, Apostolski S, Evoli A, et al. Guidelines for treatment of autoimmune neuromuscular transmission disorders. Eur J Neurol 2010 [Epub ahead of print].
12. Hart KI, Sharshar T, Sathasivam S. Immunosuppressant drugs for myasthenia gravis. J Neurol Neurosurg Psychiatry 2009; 80:5-6.
13. Ponseti JM, Gamez J, Azem J, et al. Tacrolimus for myasthenia gravis: a clinical study of 212 patients. Ann N Y Acad Sci 2008; 1132:254-263.
14. Nagane Y, Suzuki S, Suzuki N, Utsugisawa K. Factors associated with response to calcineurin inhibitors in myasthenia gravis. Muscle Nerve 2010; 41:212-218.
15. The Muscle Study Group. A trial of mycophenolate mofetil with prednisone as initial immunotherapy in myasthenia gravis. Neurology 2008; 71:394-399.
16. Sanders DB, Hart IK, Mantegazza R, et al. An international, phase III, randomized trial of mycophenolate mofetil in myasthenia gravis. Neurology 2008; 71:400-406.
17. Hehir MK, Burns TM, Alpers J, et al. Mycophenolate mofetil in AChR-antibodypositive myasthenia gravis: outcomes in 102 patients. Muscle Nerve 2010; 41:593-598.
18. Zebardast N, Patwa HS, Novella SP, Goldstein JM. Rituximab in the management of refractory myasthenia gravis. Muscle Nerve 2010; 41:375-378.
19. Argov Z, McKee D, Agus S, et al. Treatment of human myasthenia gravis with oral antisense suppression of acetylcholinesterase. Neurology 2007; 69:699-700.
20. Argov Z. Management of myasthenic conditions: nonimmune issues. Curr Opin Neurol 2009; 22:493-497.
21. Oh SJ, Claussen GC, Hatanaka Y, Morgan ML. 3,4-Diaminopyridine is more effective than placebo in a randomized, double-blind, cross-over drug study in LEMS. Muscle Nerve 2009; 40:795-800.
22. Sanders DB, Massey JM, Sanders LL, Edwards LJ. A randomized trial of 3,4-diaminopyridine in Lambert-Eaton myasthenic syndrome. Neurology 2000; 54:603-607.
23. Bestue-Cardiel M, de Cabezon-Alvarez AS, Capablo-Liesa JL, et al. Congenital endplate acetylcholinesterase deficiency responsive to ephedrine. Neurology 2005; 65:144-146.
24. Beeson D, Higuchi O, Palace J, et al. Dok-7 mutations underlie a neuromuscular junction synaptopathy. Science 2006; 313:1975-1978.
25. Schara U, Barisic N, Deschauer M, et al. Ephedrine therapy in eight patients with congenital myasthenic syndrome due to DOK7 mutations. Neuromuscul Disord 2009; 19:828-832.
26. Lashley D, Palace J, Jayawant S, et al. Ephedrine treatment in congenital myasthenic syndrome due to mutations in DOK7. Neurology 2010; 74:1517-1523.
27. Harper CM, Fukodome T, Engel AG. Treatment of slow-channel congenital myasthenic syndrome with fluoxetine. Neurology 2003; 60:1710-1713.
28. Harper CM, Engel AG. Quinidine sulfate therapy for the slow-channel congenital myasthenic syndrome. Neurology 1998; 43:480-484.
29. Amato AA, Barohn RJ. Evaluation and treatment of inflammatory myopathies. J Neurol Neurosurg Psychiatry 2009; 80:1060-1068.
30. Amato AA, Barohn RJ. Inclusion body myositis: old and new concepts. J Neurol Neurosurg Psychiatry 2009; 80:1186-1193.
31. Dalakas MC, Rakocevic G, Schmidt J, et al. Effect of alemtuzumab (CAMPATH 1-H) in patients with inclusion-body myositis. Brain 2009; 132:1536-1544.
32. Mendell JR, Moxley RT, Griggs RC, et al. Randomized, double-blind six-month trial of prednisone in Duchenne;s muscular dystrophy. N Engl J Med 1989; 320:1592-1597.
33. Bushby K, Finkel R, Birnkrant DJ, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol 2010; 9:77-93.
34. Rodino-Klapac LR, Lee J-S, Mulligan RC, et al. Lack of toxicity of alphasarcoglycan overexpression supports clinical gene transfer trial in LGMD2D. Neurology 2008; 71:240-247.
35. Mendell JR, Rodino-Klapac LR, Rosales-Quintero X, et al. Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins. Ann Neurol 2009; 66:290-297.
36. van Deutekom JC, Janson AA, Ginjaar IB, et al. Local dystrophin restoration with antisense oligonucleotide PRO051. N Engl J Med 2007; 357:2677-2686.
37. Kinali M, Arechavala-Gomeza V, Feng L, et al. Local restoration of dystrophin expression with morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study. Lancet Neurol 2009; 8:918-928.
38. Yokota T, Takeda S, Lu Q-L, et al. A renaissance for antisense oligonucleotide drugs in neurology. Arch Neurol 2009; 66:32-38.