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104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07).
Schoser B, Kishnani PS, Bratkovic D, Byrne BJ, Claeys KG, Díaz-Manera J, Laforêt P, Roberts M, Toscano A, van der Ploeg AT, Castelli J, Goldman M, Holdbrook F, Sitaraman Das S, Wasfi Y, Mozaffar T; ATB200-07 Study Group. Schoser B, et al. J Neurol. 2024 Feb 28. doi: 10.1007/s00415-024-12236-0. Online ahead of print. J Neurol. 2024. PMID: 38418563
This ongoing open-label extension (OLE; NCT04138277) evaluates long-term safety and efficacy of cipa + mig. Outcomes include 6-min walk distance (6MWD), forced vital capacity (FVC), creatine kinase (CK) and hexose tetrasaccharide (Hex4) levels, patient-reported outcomes an …
This ongoing open-label extension (OLE; NCT04138277) evaluates long-term safety and efficacy of cipa + mig. Outcomes include 6-min wa …
Delineating and clarifying the concept of self-care monitoring: a concept analysis.
Johnsson N, Strandberg S, Tuvesson H, Fagerström C, Ekstedt M, Lindberg C. Johnsson N, et al. Among authors: lindberg c. Int J Qual Stud Health Well-being. 2023 Dec;18(1):2241231. doi: 10.1080/17482631.2023.2241231. Int J Qual Stud Health Well-being. 2023. PMID: 37506372 Free PMC article. Review.
A descriptive definition was constructed and presented with exemplars to encourage practice of the concept in various healthcare settings and could be of relevance to people with chronic illnesses or other long-term conditions....
A descriptive definition was constructed and presented with exemplars to encourage practice of the concept in various healthcare settings an …
Efficacy and Safety of Rituximab for New-Onset Generalized Myasthenia Gravis: The RINOMAX Randomized Clinical Trial.
Piehl F, Eriksson-Dufva A, Budzianowska A, Feresiadou A, Hansson W, Hietala MA, Håkansson I, Johansson R, Jons D, Kmezic I, Lindberg C, Lindh J, Lundin F, Nygren I, Punga AR, Press R, Samuelsson K, Sundström P, Wickberg O, Brauner S, Frisell T. Piehl F, et al. Among authors: lindberg c. JAMA Neurol. 2022 Nov 1;79(11):1105-1112. doi: 10.1001/jamaneurol.2022.2887. JAMA Neurol. 2022. PMID: 36121672 Free PMC article.
CONCLUSIONS AND RELEVANCE: A single dose of 500 mg of rituximab was associated with greater probability of minimal MG manifestations and reduced need of rescue medications compared with placebo. Further studies are needed to address long-term benefit-risk balance with this …
CONCLUSIONS AND RELEVANCE: A single dose of 500 mg of rituximab was associated with greater probability of minimal MG manifestations and red …
Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial.
Schoser B, Roberts M, Byrne BJ, Sitaraman S, Jiang H, Laforêt P, Toscano A, Castelli J, Díaz-Manera J, Goldman M, van der Ploeg AT, Bratkovic D, Kuchipudi S, Mozaffar T, Kishnani PS; PROPEL Study Group. Schoser B, et al. Lancet Neurol. 2021 Dec;20(12):1027-1037. doi: 10.1016/S1474-4422(21)00331-8. Lancet Neurol. 2021. PMID: 34800400 Clinical Trial.
INTERPRETATION: Cipaglucosidase alfa plus miglustat did not achieve statistical superiority to alglucosidase alfa plus placebo for improving 6-min walk distance in our overall population of patients with late-onset Pompe disease. Further studies should investigate the longer-t …
INTERPRETATION: Cipaglucosidase alfa plus miglustat did not achieve statistical superiority to alglucosidase alfa plus placebo for improving …
Recurrent Neovascular Age-Related Macular Degeneration after Discontinuation of Vascular Endothelial Growth Factor Inhibitors Managed in a Treat-and-Extend Regimen.
Aslanis S, Amrén U, Lindberg C, Epstein D. Aslanis S, et al. Among authors: lindberg c. Ophthalmol Retina. 2022 Jan;6(1):15-20. doi: 10.1016/j.oret.2021.03.010. Epub 2021 Mar 26. Ophthalmol Retina. 2022. PMID: 33775926 Free article.
It is difficult to predict which patients will experience a recurrence, and most of these patients do not show symptoms in the early stages of reactivation. Long-term follow-up is important, and early detection of recurrent disease can improve the chances for maintained vi …
It is difficult to predict which patients will experience a recurrence, and most of these patients do not show symptoms in the early stages …
COX deficiency and leukoencephalopathy due to a novel homozygous APOPT1/COA8 mutation.
Hedberg-Oldfors C, Darin N, Thomsen C, Lindberg C, Oldfors A. Hedberg-Oldfors C, et al. Among authors: lindberg c. Neurol Genet. 2020 Jun 16;6(4):e464. doi: 10.1212/NXG.0000000000000464. eCollection 2020 Aug. Neurol Genet. 2020. PMID: 32637636 Free PMC article.
OBJECTIVE: To describe the long-term follow-up and pathogenesis in a child with leukoencephalopathy and cytochrome c oxidase (COX) deficiency due to a novel homozygous nonsense mutation in APOPT1/COA8. ...CONCLUSIONS: We describe a novel nonsense mutation in APOPT1/COA8 an …
OBJECTIVE: To describe the long-term follow-up and pathogenesis in a child with leukoencephalopathy and cytochrome c oxidase (COX) de …
Carey-Fineman-Ziter syndrome with mutations in the myomaker gene and muscle fiber hypertrophy.
Hedberg-Oldfors C, Lindberg C, Oldfors A. Hedberg-Oldfors C, et al. Among authors: lindberg c. Neurol Genet. 2018 Jul 23;4(4):e254. doi: 10.1212/NXG.0000000000000254. eCollection 2018 Aug. Neurol Genet. 2018. PMID: 30065953 Free PMC article.
OBJECTIVE: To describe the long-term clinical follow-up in 3 siblings with Carey-Fineman-Ziter syndrome (CFZS), a form of congenital myopathy with a novel mutation in the myomaker gene (MYMK). ...
OBJECTIVE: To describe the long-term clinical follow-up in 3 siblings with Carey-Fineman-Ziter syndrome (CFZS), a form of congenital …
Quantification of mobility impairment and self-assessment of stiffness in patients with myotonia congenita by the physiotherapist.
Hammarén E, Kjellby-Wendt G, Lindberg C. Hammarén E, et al. Among authors: lindberg c. Neuromuscul Disord. 2005 Oct;15(9-10):610-7. doi: 10.1016/j.nmd.2005.07.002. Neuromuscul Disord. 2005. PMID: 16084086
When considering the results, three instruments are favoured; Timed Up&Go and BorgCR10 for short, and MBS for long-term evaluations....
When considering the results, three instruments are favoured; Timed Up&Go and BorgCR10 for short, and MBS for long-term evaluatio …
Myosin storage myopathy associated with a heterozygous missense mutation in MYH7.
Tajsharghi H, Thornell LE, Lindberg C, Lindvall B, Henriksson KG, Oldfors A. Tajsharghi H, et al. Among authors: lindberg c. Ann Neurol. 2003 Oct;54(4):494-500. doi: 10.1002/ana.10693. Ann Neurol. 2003. PMID: 14520662
Our findings indicate that the mutated residue of slow/beta-cardiac MyHC is essential for the assembly of thick filaments in skeletal muscle. We propose the term myosin storage myopathy for this disease....
Our findings indicate that the mutated residue of slow/beta-cardiac MyHC is essential for the assembly of thick filaments in skeletal muscle …
Comparison of fresh and cryopreserved porcine ventral mesencephalon cells transplanted in A rat model of Parkinson's disease.
Jacoby DB, Lindberg C, Ratliff J, Wetzel K, Stewart GR, Dinsmore J. Jacoby DB, et al. Among authors: lindberg c. J Neurosci Res. 2002 Aug 1;69(3):382-96. doi: 10.1002/jnr.10297. J Neurosci Res. 2002. PMID: 12125079
In conclusion, the results indicate that it is feasible to cryopreserve porcine ventral mesencephalon cells for long-term storage of cells prior to transplantation in an animal model of Parkinson's disease....
In conclusion, the results indicate that it is feasible to cryopreserve porcine ventral mesencephalon cells for long-term storage of …
12 results