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Gene therapy with bidridistrogene xeboparvovec for limb-girdle muscular dystrophy type 2E/R4: phase 1/2 trial results.
Mendell JR, Pozsgai ER, Lewis S, Griffin DA, Lowes LP, Alfano LN, Lehman KJ, Church K, Reash NF, Iammarino MA, Sabo B, Potter R, Neuhaus S, Li X, Stevenson H, Rodino-Klapac LR. Mendell JR, et al. Among authors: griffin da. Nat Med. 2024 Jan;30(1):199-206. doi: 10.1038/s41591-023-02730-9. Epub 2024 Jan 4. Nat Med. 2024. PMID: 38177855 Free PMC article. Clinical Trial.
The 2-year safety and efficacy of bidridistrogene xeboparvovec support clinical development advancement. Further studies are necessary to confirm the long-term safety and efficacy of this gene therapy. ClinicalTrials.gov registration: NCT03652259 ....
The 2-year safety and efficacy of bidridistrogene xeboparvovec support clinical development advancement. Further studies are necessary to co …
Long-term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: A phase 1/2a nonrandomized trial.
Mendell JR, Sahenk Z, Lehman KJ, Lowes LP, Reash NF, Iammarino MA, Alfano LN, Lewis S, Church K, Shell R, Potter RA, Griffin DA, Hogan M, Wang S, Mason S, Darton E, Rodino-Klapac LR. Mendell JR, et al. Among authors: griffin da. Muscle Nerve. 2024 Jan;69(1):93-98. doi: 10.1002/mus.27955. Epub 2023 Aug 14. Muscle Nerve. 2024. PMID: 37577753 Clinical Trial.
INTRODUCTION/AIMS: Delandistrogene moxeparvovec is indicated in the United States for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. Long-term delandistrogene moxeparvo …
INTRODUCTION/AIMS: Delandistrogene moxeparvovec is indicated in the United States for the treatment of ambulatory pediatric patients aged 4 …
Systemic Delivery of Dysferlin Overlap Vectors Provides Long-Term Gene Expression and Functional Improvement for Dysferlinopathy.
Potter RA, Griffin DA, Sondergaard PC, Johnson RW, Pozsgai ER, Heller KN, Peterson EL, Lehtimäki KK, Windish HP, Mittal PJ, Albrecht DE, Mendell JR, Rodino-Klapac LR. Potter RA, et al. Among authors: griffin da. Hum Gene Ther. 2018 Jul;29(7):749-762. doi: 10.1089/hum.2017.062. Epub 2017 Jul 13. Hum Gene Ther. 2018. PMID: 28707952 Free PMC article.
Twenty years of high-resolution sea surface temperature imagery around Australia: inter-annual and annual variability.
Foster SD, Griffin DA, Dunstan PK. Foster SD, et al. Among authors: griffin da. PLoS One. 2014 Jul 2;9(7):e100762. doi: 10.1371/journal.pone.0100762. eCollection 2014. PLoS One. 2014. PMID: 24988444 Free PMC article.
We utilise satellite data and validate the statistical summary from these data to summaries of data from long-term monitoring stations and from the global drifter program. The spatially dense results, available as maps from the Australian Oceanographic Data Network's data …
We utilise satellite data and validate the statistical summary from these data to summaries of data from long-term monitoring station …
Cardiac extracorporeal life support: state of the art in 2007.
Cooper DS, Jacobs JP, Moore L, Stock A, Gaynor JW, Chancy T, Parpard M, Griffin DA, Owens T, Checchia PA, Thiagarajan RR, Spray TL, Ravishankar C. Cooper DS, et al. Among authors: griffin da. Cardiol Young. 2007 Sep;17 Suppl 2:104-15. doi: 10.1017/S1047951107001217. Cardiol Young. 2007. PMID: 18039404 Review.
Although extracorporeal membrane oxygenation has become a standard of care for many paediatric centres, its use is limited to those patients who require only short-term cardiopulmonary support. Mechanical ventricular assist devices have become standard therapy for adults w …
Although extracorporeal membrane oxygenation has become a standard of care for many paediatric centres, its use is limited to those patients …