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Publikationer i urvalet

28 träffar
Bibliografisk träff 2024-04-01

Vamorolone.

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Am J Health Syst Pharm · journal article · DOI 10.1093/ajhp/zxad303
Bibliografisk träff 2024-02-09

Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy: A Randomized Controlled Trial.

Dang UJ, Damsker JM, Guglieri M, Clemens PR, Perlman SJ, Smith EC, Horrocks I, Finkel RS, Mah JK, Deconinck N, Goemans NM, Haberlová J, Straub V, Mengle-Gaw L, Schwartz BD, Harper A, Shieh PB, De Waele L, Castro D, Yang ML, Ryan MM, McDonald CM, Tulinius M, Webster RI, Mcmillan HJ, Kuntz N, Rao VK, Baranello G, Spinty S, Childs AM, Sbrocchi AM, Selby KA, Monduy M, Nevo Y, Vilchez JJ, Nascimento-Osorio A, Niks EH, De Groot IJM, Katsalouli M, Van Den Anker JN, Ward LM, Leinonen M, D'Alessandro AL, Hoffman EP.

Neurology · research support, non-u.s. gov't; research-article; randomized controlled trial; journal article; research support, n.i.h., extramural · DOI 10.1212/wnl.0000000000208112
Bibliografisk träff 2024-01-01

Comparison of pharmaceutical properties and biological activities of prednisolone, deflazacort, and vamorolone in DMD disease models.

Liu G, Lipari P, Mollin A, Jung S, Teplova I, Li W, Ying L, More V, Lennox W, Yeh S, McGann E, Moon YC, Rice C, Huarte E, Gruszka B, Ray B, Goodwin E, Buckendahl P, Yurkow E, Braughton B, Narasimhan J, Welch E, Voronin G, Weetall M.

Hum Mol Genet · research support, non-u.s. gov't; research-article; journal article · DOI 10.1093/hmg/ddad173
Bibliografisk träff 2022-10-01

Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.

Guglieri M, Clemens PR, Perlman SJ, Smith EC, Horrocks I, Finkel RS, Mah JK, Deconinck N, Goemans N, Haberlova J, Straub V, Mengle-Gaw LJ, Schwartz BD, Harper AD, Shieh PB, De Waele L, Castro D, Yang ML, Ryan MM, McDonald CM, Tulinius M, Webster R, McMillan HJ, Kuntz NL, Rao VK, Baranello G, Spinty S, Childs AM, Sbrocchi AM, Selby KA, Monduy M, Nevo Y, Vilchez-Padilla JJ, Nascimento-Osorio A, Niks EH, de Groot IJM, Katsalouli M, James MK, van den Anker J, Damsker JM, Ahmet A, Ward LM, Jaros M, Shale P, Dang UJ, Hoffman EP.

JAMA Neurol · research support, non-u.s. gov't; research-article; multicenter study; randomized controlled trial; journal article; research support, n.i.h., extramural · DOI 10.1001/jamaneurol.2022.2480
Bibliografisk träff 2022-01-04

Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial.

Mah JK, Clemens PR, Guglieri M, Smith EC, Finkel RS, Tulinius M, Nevo Y, Ryan MM, Webster R, Castro D, Kuntz NL, McDonald CM, Damsker JM, Schwartz BD, Mengle-Gaw LJ, Jackowski S, Stimpson G, Ridout DA, Ayyar-Gupta V, Baranello G, Manzur AY, Muntoni F, Gordish-Dressman H, Leinonen M, Ward LM, Hoffman EP, Dang UJ, NorthStar UK Network and CINRG DNHS Investigators.

JAMA Netw Open · clinical trial; research support, non-u.s. gov't; research-article; research support, u.s. gov't, non-p.h.s.; journal article; research support, n.i.h., extramural · DOI 10.1001/jamanetworkopen.2021.44178
Bibliografisk träff 2020-09-21

Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study.

Smith EC, Conklin LS, Hoffman EP, Clemens PR, Mah JK, Finkel RS, Guglieri M, Tulinius M, Nevo Y, Ryan MM, Webster R, Castro D, Kuntz NL, Kerchner L, Morgenroth LP, Arrieta A, Shimony M, Jaros M, Shale P, Gordish-Dressman H, Hagerty L, Dang UJ, Damsker JM, Schwartz BD, Mengle-Gaw LJ, McDonald CM, CINRG VBP15 and DNHS Investigators.

PLoS Med · clinical trial, phase ii; research support, non-u.s. gov't; research-article; multicenter study; journal article; research support, n.i.h., extramural · DOI 10.1371/journal.pmed.1003222
Bibliografisk träff 2019-08-26

Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function.

Hoffman EP, Schwartz BD, Mengle-Gaw LJ, Smith EC, Castro D, Mah JK, McDonald CM, Kuntz NL, Finkel RS, Guglieri M, Bushby K, Tulinius M, Nevo Y, Ryan MM, Webster R, Smith AL, Morgenroth LP, Arrieta A, Shimony M, Siener C, Jaros M, Shale P, McCall JM, Nagaraju K, van den Anker J, Conklin LS, Cnaan A, Gordish-Dressman H, Damsker JM, Clemens PR, Cooperative International Neuromuscular Research Group.

Neurology · clinical trial; research support, non-u.s. gov't; research-article; journal article; research support, n.i.h., extramural · DOI 10.1212/wnl.0000000000008168
Bibliografisk träff 2018-09-13

Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug.

Conklin LS, Damsker JM, Hoffman EP, Jusko WJ, Mavroudis PD, Schwartz BD, Mengle-Gaw LJ, Smith EC, Mah JK, Guglieri M, Nevo Y, Kuntz N, McDonald CM, Tulinius M, Ryan MM, Webster R, Castro D, Finkel RS, Smith AL, Morgenroth LP, Arrieta A, Shimony M, Jaros M, Shale P, McCall JM, Hathout Y, Nagaraju K, van den Anker J, Ward LM, Ahmet A, Cornish MR, Clemens PR.

Pharmacol Res · clinical trial, phase ii; research support, non-u.s. gov't; research-article; multicenter study; research support, u.s. gov't, non-p.h.s.; journal article; research support, n.i.h., extramural · DOI 10.1016/j.phrs.2018.09.007
Bibliografisk träff 2018-03-08

Phase 1 trial of vamorolone, a first-in-class steroid, shows improvements in side effects via biomarkers bridged to clinical outcomes.

Hoffman EP, Riddle V, Siegler MA, Dickerson D, Backonja M, Kramer WG, Nagaraju K, Gordish-Dressman H, Damsker JM, McCall JM.

Steroids · research support, non-u.s. gov't; research-article; clinical trial, phase i; randomized controlled trial; journal article; research support, n.i.h., extramural · DOI 10.1016/j.steroids.2018.02.010
Bibliografisk träff 2013-09-09

VBP15, a novel anti-inflammatory and membrane-stabilizer, improves muscular dystrophy without side effects.

Heier CR, Damsker JM, Yu Q, Dillingham BC, Huynh T, Van der Meulen JH, Sali A, Miller BK, Phadke A, Scheffer L, Quinn J, Tatem K, Jordan S, Dadgar S, Rodriguez OC, Albanese C, Calhoun M, Gordish-Dressman H, Jaiswal JK, Connor EM, McCall JM, Hoffman EP, Reeves EK, Nagaraju K.

EMBO Mol Med · research support, non-u.s. gov't; research-article; research support, u.s. gov't, non-p.h.s.; journal article; research support, n.i.h., extramural · DOI 10.1002/emmm.201302621
Bibliografisk träff 2013-05-07

VBP15, a glucocorticoid analogue, is effective at reducing allergic lung inflammation in mice.

Damsker JM, Dillingham BC, Rose MC, Balsley MA, Heier CR, Watson AM, Stemmy EJ, Jurjus RA, Huynh T, Tatem K, Uaesoontrachoon K, Berry DM, Benton AS, Freishtat RJ, Hoffman EP, McCall JM, Gordish-Dressman H, Constant SL, Reeves EK, Nagaraju K.

PLoS One · research support, non-u.s. gov't; research-article; research support, u.s. gov't, non-p.h.s.; journal article; research support, n.i.h., extramural · DOI 10.1371/journal.pone.0063871