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SMA Related Publications by PNCR Members

Montes J, Gordon AM, Pandya S, De Vivo DC, Kaufmann P. Clinical Outcome Measures in Spinal Muscular Atrophy. Journal of Child Neurology. 2009, 24:968-978.

Sproule DM, Montes J, Montgomery M, Battista V, Koenigsberger D, Shen W, Punyanitya M, De Vivo DC, Kaufmann P. Increased fat mass and high incidence of overweight despite low body mass index in patients with spinal muscular atrophy. Neuromuscul Disord. 2009, Jun;19(6):391-6..

Bönnemann CG, Finkel RS. Linking SMN to SMA: an assay for the rescuer. Neurology. 2008 Nov;71(22):1752-1753.

Finkel RS, Hynan LS, Glanzman AM, Owens H, Nelson, L, Cone SR, Campbell SK, Iannaccone ST, and the AmSMART Group. The Test of Infant Motor performance: reliability in spinal muscular atrophy type 1. Pediatric Physical Therapy. 2008 Fall;20(3):242-6.

Kaufmann P, Iannaccone ST. Clinical trials in spinal muscular atrophy. Phys Med Rehabil Clin N Am. 2008 Aug;19(3):653-60, xii.

Oskoui M, Kaufmann P. Spinal muscular atrophy. Neurotherapeutics. 2008 Oct;5(4):499-506.

Darras BT, Kang PB. Clinical trials in spinal muscular atrophy. Curr Opin Pediatr. 2007 Dec;19(6):675-9.

Kaufmann P, Finkel R. Learning to walk: challenges for spinal muscular atrophy clinical trials. Neurology. 2007 Jan;68(1):11-12.

Kaufmann P, Muntoni F; International Coordinating Committee for SMA Subcommittee on SMA Clinical Trial Design. Issues in SMA clinical trial design. The International Coordinating Committee (ICC) for SMA Subcommittee on SMA Clinical Trial Design. Neuromuscul Disord. 2007 Jun;17(6):499-505. Epub 2007 Feb 14.

O'Hagen JM, Glanzman AM, McDermott MP, Ryan PA, Flickinger J, Quigley J, Riley S, Sanborn E, Irvine C, Martens WB, Annis C, Tawil R, Oskoui M, Darras BT, Finkel RS, De Vivo DC. An expanded version of the Hammersmith Functional Motor Scale for SMA II and III patients. Neuromuscul Disord. 2007 Oct;17(9-10):693-7. Epub 2007 Jul 19.

Oskoui M, Levy G, Garland CJ, Gray JM, O'Hagen J, De Vivo DC, Kaufmann P. The changing natural history of spinal muscular atrophy type 1. Neurology. 2007 Nov 13;69(20):1931-6.

Wang CH, Finkel RS, Bertini ES, Schroth M, Simonds A, Wong B, Aloysius A, Morrison L, Main M, Crawford TO, Trela A; Participants of the Internatioinal Conference on SMA Standard of Care. Consensus statement for standard of care in spinal muscular atrophy. J Child Neurol. 2007 Aug;22(8):1027-49.