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Defining consensus opinion to develop randomised controlled trials in rare diseases using Bayesian design: An example of a proposed trial of adalimumab versus pamidronate for children with CNO/CRMO.
Ramanan AV, Hampson LV, Lythgoe H, Jones AP, Hardwick B, Hind H, Jacobs B, Vasileiou D, Wadsworth I, Ambrose N, Davidson J, Ferguson PJ, Herlin T, Kavirayani A, Killeen OG, Compeyrot-Lacassagne S, Laxer RM, Roderick M, Swart JF, Hedrich CM, Beresford MW. Ramanan AV, et al. Among authors: jacobs b. PLoS One. 2019 Jun 5;14(6):e0215739. doi: 10.1371/journal.pone.0215739. eCollection 2019. PLoS One. 2019. PMID: 31166977 Free PMC article.
Bone health and SATB2-associated syndrome.
Zarate YA, Steinraths M, Matthews A, Smith WE, Sun A, Wilson LC, Brain C, Allgove J, Jacobs B, Fish JL, Powell CM, Wasserman WW, van Karnebeek CD, Wakeling EL, Ma NS. Zarate YA, et al. Among authors: jacobs b. Clin Genet. 2018 Mar;93(3):588-594. doi: 10.1111/cge.13121. Epub 2017 Dec 27. Clin Genet. 2018. PMID: 28787087 Review.
Guillain-Barré syndrome.
Leonhard SE, Papri N, Querol L, Rinaldi S, Shahrizaila N, Jacobs BC. Leonhard SE, et al. Among authors: jacobs bc. Nat Rev Dis Primers. 2024 Dec 19;10(1):97. doi: 10.1038/s41572-024-00580-4. Nat Rev Dis Primers. 2024. PMID: 39702645
Challenges in the identification and quantification of an unknown impurity in chenodeoxycholic acid drug substance.
Bouwhuis N, Polak Y, Schimmel AM, Bijleveld YA, Giera MA, Heijink M, Vaz FM, Bootsma AH, Berg-Lammers LAT, Swart NEL, Hollak CEM, Jacobs BAW, Kemper EM. Bouwhuis N, et al. Among authors: jacobs baw. Eur J Pharm Sci. 2024 Dec 4;205:106979. doi: 10.1016/j.ejps.2024.106979. Online ahead of print. Eur J Pharm Sci. 2024. PMID: 39643128 Free article.
Long-Term Follow Up in Anti-Contactin-1 Autoimmune Nodopathy.
Caballero-Ávila M, Martín-Aguilar L, Pascual-Goñi E, Michael MR, Koel-Simmelink MJA, Höftberger R, Wanschitz J, Alonso-Jiménez A, Armangué T, Baars AE, Carbayo Á, Castek B, Collet-Vidiella R, De Winter J, Del Real MÁ, Delmont E, Diamanti L, Doneddu PE, Hiew FL, Gallardo E, Gonzalez A, Grinzinger S, Horga A, Iglseder S, Jacobs BC, Jauregui A, Killestein J, Pozza EL, Martínez-Martínez L, Nobile-Orazio E, Ortiz N, Pérez-Pérez H, Poppert KN, Ripellino P, Roche JC, Rodriguez de Rivera FJ, Rostasy K, Sparasci D, Tejada-Illa C, Teunissen CCE, Vegezzi E, Xuclà-Ferrarons T, Zach F, Wieske L, Eftimov F, Lleixà C, Querol L. Caballero-Ávila M, et al. Among authors: jacobs bc. Ann Neurol. 2024 Nov 27. doi: 10.1002/ana.27142. Online ahead of print. Ann Neurol. 2024. PMID: 39601182
1,860 results