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Provisional practice recommendation for the management of myopathy in VCP-associated multisystem proteinopathy.
Roy B, Peck A, Evangelista T, Pfeffer G, Wang L, Diaz-Manera J, Korb M, Wicklund MP, Milone M, Freimer M, Kushlaf H, Villar-Quiles RN, Stojkovic T, Needham M, Palmio J, Lloyd TE, Keung B, Mozaffar T, Weihl CC, Kimonis V. Roy B, et al. Among authors: wang l. Ann Clin Transl Neurol. 2023 May;10(5):686-695. doi: 10.1002/acn3.51760. Epub 2023 Apr 7. Ann Clin Transl Neurol. 2023. PMID: 37026610 Free PMC article. Review.
Development of a standard of care for patients with valosin-containing protein associated multisystem proteinopathy.
Korb M, Peck A, Alfano LN, Berger KI, James MK, Ghoshal N, Healzer E, Henchcliffe C, Khan S, Mammen PPA, Patel S, Pfeffer G, Ralston SH, Roy B, Seeley WW, Swenson A, Mozaffar T, Weihl C, Kimonis V; VCP Standards of Care Working Group. Korb M, et al. Orphanet J Rare Dis. 2022 Jan 29;17(1):23. doi: 10.1186/s13023-022-02172-5. Orphanet J Rare Dis. 2022. PMID: 35093159 Free PMC article. Review.
Current status of clinical outcome measures in inclusion body myositis: a systematised review.
Roy B, Lucchini M, Lilleker JB, Goyal NA, Naddaf E, Adler B, Alfano LN, Malandraki GA, Focht Garand KL, Mochel D, Badrising U, Machado PM, Pagkatipunan R, Ramdharry G, Wang L, Funaro MC, Schmidt J, Kushlaf H, Schiopu E, Stipancic K, Goyal N, d'Alessandro M, Conticini E, Cruz-Coble B, Lloyd TE; International Myositis Assessment and Clinical Studies (IMACS) Inclusion Body Myositis Scientific Interest Group. Roy B, et al. Among authors: wang l. Clin Exp Rheumatol. 2023 Mar;41(2):370-378. doi: 10.55563/clinexprheumatol/ifacv3. Epub 2023 Feb 3. Clin Exp Rheumatol. 2023. PMID: 36762744 Free article. Review.
Design and Statistical Innovations in a Platform Trial for Amyotrophic Lateral Sclerosis.
Quintana M, Saville BR, Vestrucci M, Detry MA, Chibnik L, Shefner J, Berry JD, Chase M, Andrews J, Sherman AV, Yu H, Drake K, Cudkowicz M, Paganoni S, Macklin EA; HEALEY ALS Platform Trial Study Group. Quintana M, et al. Ann Neurol. 2023 Sep;94(3):547-560. doi: 10.1002/ana.26714. Epub 2023 Jun 22. Ann Neurol. 2023. PMID: 37245090
Safety and efficacy of losmapimod in facioscapulohumeral muscular dystrophy (ReDUX4): a randomised, double-blind, placebo-controlled phase 2b trial.
Tawil R, Wagner KR, Hamel JI, Leung DG, Statland JM, Wang LH, Genge A, Sacconi S, Lochmüller H, Reyes-Leiva D, Diaz-Manera J, Alonso-Perez J, Muelas N, Vilchez JJ, Pestronk A, Gibson S, Goyal NA, Hayward LJ, Johnson N, LoRusso S, Freimer M, Shieh PB, Subramony SH, van Engelen B, Kools J, Leinhard OD, Widholm P, Morabito C, Moxham CM, Cadavid D, Mellion ML, Odueyungbo A, Tracewell WG, Accorsi A, Ronco L, Gould RJ, Shoskes J, Rojas LA, Jiang JG. Tawil R, et al. Among authors: wang lh. Lancet Neurol. 2024 May;23(5):477-486. doi: 10.1016/S1474-4422(24)00073-5. Lancet Neurol. 2024. PMID: 38631764 Clinical Trial.
Defining SOD1 ALS natural history to guide therapeutic clinical trial design.
Bali T, Self W, Liu J, Siddique T, Wang LH, Bird TD, Ratti E, Atassi N, Boylan KB, Glass JD, Maragakis NJ, Caress JB, McCluskey LF, Appel SH, Wymer JP, Gibson S, Zinman L, Mozaffar T, Callaghan B, McVey AL, Jockel-Balsarotti J, Allred P, Fisher ER, Lopate G, Pestronk A, Cudkowicz ME, Miller TM. Bali T, et al. Among authors: wang lh. J Neurol Neurosurg Psychiatry. 2017 Feb;88(2):99-105. doi: 10.1136/jnnp-2016-313521. Epub 2016 Jun 3. J Neurol Neurosurg Psychiatry. 2017. PMID: 27261500 Free PMC article.
Immunophenotyping of Inclusion Body Myositis Blood T and NK Cells.
Goyal NA, Coulis G, Duarte J, Farahat PK, Mannaa AH, Cauchii J, Irani T, Araujo N, Wang L, Wencel M, Li V, Zhang L, Greenberg SA, Mozaffar T, Villalta SA. Goyal NA, et al. Among authors: wang l. Neurology. 2022 Mar 29;98(13):e1374-e1383. doi: 10.1212/WNL.0000000000200013. Epub 2022 Feb 7. Neurology. 2022. PMID: 35131904 Free PMC article.
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