Clinical trial NCT05284006 - References

Morquio A Syndrome — Non-invasive Functional Assessment and Pathogenesis of Morquio A

Citation(s)

•   Baratela WA, Bober MB, Thacker MM, Belthur MV, Oto M, Rogers KJ, Mackenzie WG
  Cervicothoracic myelopathy in children with Morquio syndrome A: a report of 4 cases. J Pediatr Orthop. 2014 Mar;34(2):223-8. doi: 10.1097/BPO.0000000000000074.
•   Dede O, Thacker MM, Rogers KJ, Oto M, Belthur MV, Baratela W, Mackenzie WG
  Upper cervical fusion in children with Morquio syndrome: intermediate to long-term results. J Bone Joint Surg Am. 2013 Jul 3;95(13):1228-34. doi: 10.2106/JBJS.J.01135.
•   Doherty C, Stapleton M, Piechnik M, Mason RW, Mackenzie WG, Yamaguchi S, Kobayashi H, Suzuki Y, Tomatsu S
  Effect of enzyme replacement therapy on the growth of patients with Morquio A. J Hum Genet. 2019 Jul;64(7):625-635. doi: 10.1038/s10038-019-0604-6. Epub 2019 Apr 24.
•   Montano AM, Tomatsu S, Brusius A, Smith M, Orii T
  Growth charts for patients affected with Morquio A disease. Am J Med Genet A. 2008 May 15;146A(10):1286-95. doi: 10.1002/ajmg.a.32281.
•   Montano AM, Tomatsu S, Gottesman GS, Smith M, Orii T
  International Morquio A Registry: clinical manifestation and natural course of Morquio A disease. J Inherit Metab Dis. 2007 Apr;30(2):165-74. doi: 10.1007/s10545-007-0529-7. Epub 2007 Mar 8.
•   Pizarro C, Davies RR, Theroux M, Spurrier EA, Averill LW, Tomatsu S
  Surgical Reconstruction for Severe Tracheal Obstruction in Morquio A Syndrome. Ann Thorac Surg. 2016 Oct;102(4):e329-31. doi: 10.1016/j.athoracsur.2016.02.113.
•   Sitoula P, Mackenzie WG, Shah SA, Thacker M, Ditro C, Holmes L Jr, Campbell JW, Rogers KJ
  Occipitocervical fusion in skeletal dysplasia: a new surgical technique. Spine (Phila Pa 1976). 2014 Jul 1;39(15):E912-8. doi: 10.1097/BRS.0000000000000381.
•   Tomatsu S, Montano AM, Oguma T, Dung VC, Oikawa H, de Carvalho TG, Gutierrez ML, Yamaguchi S, Suzuki Y, Fukushi M, Kida K, Kubota M, Barrera L, Orii T
  Validation of keratan sulfate level in mucopolysaccharidosis type IVA by liquid chromatography-tandem mass spectrometry. J Inherit Metab Dis. 2010 Dec;33 Suppl 3:S35-42. doi: 10.1007/s10545-009-9013-x. Epub 2010 Jan 27.
•   Tomatsu S, Okamura K, Taketani T, Orii KO, Nishioka T, Gutierrez MA, Velez-Castrillon S, Fachel AA, Grubb JH, Cooper A, Thornley M, Wraith E, Barrera LA, Giugliani R, Schwartz IV, Frenking GS, Beck M, Kircher SG, Paschke E, Yamaguchi S, Ullrich K, Isogai K, Suzuki Y, Orii T, Kondo N, Creer M, Noguchi A
  Development and testing of new screening method for keratan sulfate in mucopolysaccharidosis IVA. Pediatr Res. 2004 Apr;55(4):592-7. doi: 10.1203/01.PDR.0000113767.60140.E9. Epub 2004 Jan 7.
•   Whyte MP, Fujita KP, Moseley S, Thompson DD, McAlister WH
  Validation of a Novel Scoring System for Changes in Skeletal Manifestations of Hypophosphatasia in Newborns, Infants, and Children: The Radiographic Global Impression of Change Scale. J Bone Miner Res. 2018 May;33(5):868-874. doi: 10.1002/jbmr.3377. Epub 2018 Feb 14.

Non-invasive Functional Assessment and Pathogenesis of Morquio A (NIFAMA)

Details for clinical trial NCT05284006