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Items: 1 to 20 of 87

1.

Placebo-controlled crossover assessment of mecasermin for the treatment of Rett syndrome.

O'Leary HM, Kaufmann WE, Barnes KV, Rakesh K, Kapur K, Tarquinio DC, Cantwell NG, Roche KJ, Rose SA, Walco AC, Bruck NM, Bazin GA, Holm IA, Alexander ME, Swanson LC, Baczewski LM, Poon C, Mayor Torres JM, Nelson CA 3rd, Sahin M.

Ann Clin Transl Neurol. 2018 Jan 31;5(3):323-332. doi: 10.1002/acn3.533. eCollection 2018 Mar. Erratum in: Ann Clin Transl Neurol. 2018 Jul 13;5(7):895. Poon C [added].

2.

Anxiety-like behavior in Rett syndrome: characteristics and assessment by anxiety scales.

Barnes KV, Coughlin FR, O'Leary HM, Bruck N, Bazin GA, Beinecke EB, Walco AC, Cantwell NG, Kaufmann WE.

J Neurodev Disord. 2015;7(1):30. doi: 10.1186/s11689-015-9127-4. Epub 2015 Sep 15.

4.

Social impairments in Rett syndrome: characteristics and relationship with clinical severity.

Kaufmann WE, Tierney E, Rohde CA, Suarez-Pedraza MC, Clarke MA, Salorio CF, Bibat G, Bukelis I, Naram D, Lanham DC, Naidu S.

J Intellect Disabil Res. 2012 Mar;56(3):233-47. doi: 10.1111/j.1365-2788.2011.01404.x. Epub 2011 Mar 8.

PMID:
21385260
5.

Safety, pharmacokinetics, and preliminary assessment of efficacy of mecasermin (recombinant human IGF-1) for the treatment of Rett syndrome.

Khwaja OS, Ho E, Barnes KV, O'Leary HM, Pereira LM, Finkelstein Y, Nelson CA 3rd, Vogel-Farley V, DeGregorio G, Holm IA, Khatwa U, Kapur K, Alexander ME, Finnegan DM, Cantwell NG, Walco AC, Rappaport L, Gregas M, Fichorova RN, Shannon MW, Sur M, Kaufmann WE.

Proc Natl Acad Sci U S A. 2014 Mar 25;111(12):4596-601. doi: 10.1073/pnas.1311141111. Epub 2014 Mar 12.

6.

A phase 1/2, open-label assessment of the safety, tolerability, and efficacy of transdermal cannabidiol (ZYN002) for the treatment of pediatric fragile X syndrome.

Heussler H, Cohen J, Silove N, Tich N, Bonn-Miller MO, Du W, O'Neill C, Sebree T.

J Neurodev Disord. 2019 Aug 2;11(1):16. doi: 10.1186/s11689-019-9277-x.

7.

Double-blind, randomized, placebo-controlled study of trofinetide in pediatric Rett syndrome.

Glaze DG, Neul JL, Kaufmann WE, Berry-Kravis E, Condon S, Stoms G, Oosterholt S, Della Pasqua O, Glass L, Jones NE, Percy AK; Rett 002 Study Group.

Neurology. 2019 Apr 16;92(16):e1912-e1925. doi: 10.1212/WNL.0000000000007316. Epub 2019 Mar 27.

8.

Duloxetine 60 mg once-daily in the treatment of painful physical symptoms in patients with major depressive disorder.

Brannan SK, Mallinckrodt CH, Brown EB, Wohlreich MM, Watkin JG, Schatzberg AF.

J Psychiatr Res. 2005 Jan;39(1):43-53.

PMID:
15504423
9.

Effects of milnacipran on neurocognition, pain, and fatigue in fibromyalgia: a 13-week, randomized, placebo-controlled, crossover trial.

Kim JL, Rele S, Marks DM, Masand PS, Yerramsetty P, Millet RA, Keefe RS, Patkar AA.

Prim Care Companion CNS Disord. 2013;15(6). pii: PCC.13m01555. doi: 10.4088/PCC.13m01555. Epub 2014 Dec 26.

10.

A double-blind, multicenter trial comparing duloxetine with placebo in the treatment of fibromyalgia patients with or without major depressive disorder.

Arnold LM, Lu Y, Crofford LJ, Wohlreich M, Detke MJ, Iyengar S, Goldstein DJ.

Arthritis Rheum. 2004 Sep;50(9):2974-84.

11.

Effects of creatine supplementation in Rett syndrome: a randomized, placebo-controlled trial.

Freilinger M, Dunkler D, Lanator I, Item CB, Mühl A, Fowler B, Bodamer OA.

J Dev Behav Pediatr. 2011 Jul-Aug;32(6):454-60. doi: 10.1097/DBP.0b013e31822177a8.

PMID:
21654506
12.

Safety and efficacy of deferiprone for pantothenate kinase-associated neurodegeneration: a randomised, double-blind, controlled trial and an open-label extension study.

Klopstock T, Tricta F, Neumayr L, Karin I, Zorzi G, Fradette C, Kmieć T, Büchner B, Steele HE, Horvath R, Chinnery PF, Basu A, Küpper C, Neuhofer C, Kálmán B, Dušek P, Yapici Z, Wilson I, Zhao F, Zibordi F, Nardocci N, Aguilar C, Hayflick SJ, Spino M, Blamire AM, Hogarth P, Vichinsky E.

Lancet Neurol. 2019 Jul;18(7):631-642. doi: 10.1016/S1474-4422(19)30142-5.

PMID:
31202468
13.
14.

A double-blind placebo-controlled study of controlled release fluvoxamine for the treatment of generalized social anxiety disorder.

Westenberg HG, Stein DJ, Yang H, Li D, Barbato LM.

J Clin Psychopharmacol. 2004 Feb;24(1):49-55.

PMID:
14709947
15.

Illness Severity, Social and Cognitive Ability, and EEG Analysis of Ten Patients with Rett Syndrome Treated with Mecasermin (Recombinant Human IGF-1).

Pini G, Congiu L, Benincasa A, DiMarco P, Bigoni S, Dyer AH, Mortimer N, Della-Chiesa A, O'Leary S, McNamara R, Mitchell KJ, Gill M, Tropea D.

Autism Res Treat. 2016;2016:5073078. doi: 10.1155/2016/5073078. Epub 2016 Jan 26.

17.

Effects of guanfacine extended release on oppositional symptoms in children aged 6-12 years with attention-deficit hyperactivity disorder and oppositional symptoms: a randomized, double-blind, placebo-controlled trial.

Connor DF, Findling RL, Kollins SH, Sallee F, López FA, Lyne A, Tremblay G.

CNS Drugs. 2010 Sep;24(9):755-68. doi: 10.2165/11537790-000000000-00000.

PMID:
20806988
18.

Mavoglurant in adolescents with fragile X syndrome: analysis of Clinical Global Impression-Improvement source data from a double-blind therapeutic study followed by an open-label, long-term extension study.

Bailey DB Jr, Berry-Kravis E, Wheeler A, Raspa M, Merrien F, Ricart J, Koumaras B, Rosenkranz G, Tomlinson M, von Raison F, Apostol G.

J Neurodev Disord. 2016;8:1. doi: 10.1186/s11689-015-9134-5. Epub 2015 Dec 15.

20.

Randomized open-label trial of dextromethorphan in Rett syndrome.

Smith-Hicks CL, Gupta S, Ewen JB, Hong M, Kratz L, Kelley R, Tierney E, Vaurio R, Bibat G, Sanyal A, Yenokyan G, Brereton N, Johnston MV, Naidu S.

Neurology. 2017 Oct 17;89(16):1684-1690. doi: 10.1212/WNL.0000000000004515. Epub 2017 Sep 20.

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