Spina Bifida - Myelomeningocele
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Relevant Article Depot:
Myelomeningocele
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A randomized trial of prenatal versus postnatal repair of myelomeningocele
The Management of Myelomeningocele Study: full cohort 30-month pediatric outcomes
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Adzick NS, Thom EA, Spong CY, Brock JW, 3rd, Burrows PK, Johnson MP, Howell LJ, Farrell JA, Dabrowiak ME, Sutton LN et al: A randomized trial of prenatal versus postnatal repair of myelomeningocele. The New England journal of medicine 2011, 364(11):993–1004.
2. Farmer DL, Kumar P, Reynolds E, Lee SY, Powne AB, Pivetti CD, Zwienenberg M, McLennan AS, Nolta JA, Brown EG et al: Feasibility and safety of cellular therapy for in-utero repair of myelomeningocele (CuRe Trial): a first-in-human, phase 1, single-arm study. Lancet (London, England) 2026, 407(10531):867–875.
3. Cortes MS: First steps of a potential new era in fetal intervention for spina bifida. Lancet (London, England) 2026, 407(10531):827–829.
4. Theodorou CM, Stokes SC, Jackson JE, Pivetti CD, Kumar P, Yamashiro KJ, Paxton ZJ, Reynaga L, Hyllen AA, Wang A et al: Efficacy of clinical-grade human placental mesenchymal stromal cells in fetal ovine myelomeningocele repair. J Pediatr Surg 2022, 57(4):753–758.
5. Galganski LA, Kumar P, Vanover MA, Pivetti CD, Anderson JE, Lankford L, Paxton ZJ, Chung K, Lee C, Hegazi MS et al: In utero treatment of myelomeningocele with placental mesenchymal stromal cells - Selection of an optimal cell line in preparation for clinical trials. J Pediatr Surg 2020, 55(9):1941–1946.
6. Vanover M, Pivetti C, Lankford L, Kumar P, Galganski L, Kabagambe S, Keller B, Becker J, Chen YJ, Chung K et al: High density placental mesenchymal stromal cells provide neuronal preservation and improve motor function following in utero treatment of ovine myelomeningocele. J Pediatr Surg 2019, 54(1):75–79.
Sagar RL, Åström E, Chitty LS, Crowe B, David AL, DeVile C, Forsmark A, Franzen V, Hermeren G, Hill M et al: An exploratory open-label multicentre phase I/II trial evaluating the safety and efficacy of postnatal or prenatal and postnatal administration of allogeneic expanded fetal mesenchymal stem cells for the treatment of severe osteogenesis imperfecta in infants and fetuses: the BOOSTB4 trial protocol. BMJ Open 2024, 14(6):e079767.
MacKenzie TC, Frascoli M, Sper R, Lianoglou BR, Velez JG, Dvorak CC, Kharbanda S, Vichinsky E: In utero stem cell transplantation in patients with alpha thalassemia major: interim results of a phase 1 clinical trial. Blood 2020, 136:1.