Cure SMA Contributes Research Funding to Sixteen SMA Journal Articles in 2013

Originally published on December 30, 2013.

Cure SMA is proud to have contributed funding to the research published in the following journal articles in 2013. Cure SMA has invested over $55 Million in research to accelerate identification of a treatment and cure for SMA.  This includes 25 research projects in 2013 alone.  

Please see the Cure SMA Funding Facts Page for all funded projects.

Funding for these studies was awarded from Cure SMA to the following institutions: Columbia University, Indiana University, University of Utah, Ohio State University, Johns Hopkins University, University of Manitoba, Wayne State University, University of Wisconsin, Hospital Sainte-Justine, Harvard University, University of Ottawa, Northwestern University, Rutgers University, University of Southern California, Emory University, University of Cologne, and University of Milan.

Mapping The Differences In Care For 5,000 Spinal Muscular Atrophy Patients, A Survey Of 24 National Registries In North America, Australasia And Europe.  Bladen CL, Thompson R, Jackson JM, Garland C, Wegel C, Ambrosini A, Pisano P, Walter MC, Schreiber O, Lusakowska A, et al.  Journal of Neurology.

Antisense Oligonucleotide Mediated Therapy of Spinal Muscular Atrophy.  Sivanesan S, Howell MD, Didonato CJ, Singh RN.  Translational Neuroscience.

Nutritional Practices At A Glance: Spinal Muscular Atrophy Type I Nutrition Survey Findings.  Davis RH, Godshall BJ, Seffrood E, Marcus M, Lasalle BA, Wong B, Schroth MK, Swoboda KJ. Journal of Child Neurology.

Spinal Muscular Atrophy: Development And Implementation Of Potential Treatments. Arnold WD, Burghes AH. Annals of Neurology.

Dynamics Of Survival Of Motor Neuron (SMN) Protein Interaction With The Mrna-Binding Protein IMP1 Facilitates Its Trafficking Into Motor Neuron Axons. Fallini C, Rouanet JP, Donlin-Asp PG, Guo P, Zhang H, Singer RH, Rossoll W, Bassell GJ.  Developmental Neurobiology.

Rasch Analysis Of Clinical Outcome Measures In Spinal Muscular Atrophy.  Cano SJ, Mayhew A, Glanzman AM, Krosschell KJ, Swoboda KJ, Main M, Steffensen BF, Bérard C, Girardot F, Payan CA, Mercuri E, Mazzone E, Elsheikh B, Florence J, Hynan LS, Iannaccone ST, Nelson LL, Pandya S, Rose M, Scott C, Sadjadi R, Yore MA, Joyce C, Kissel JT.  Muscle and Nerve.

Genetic Circuitry Of Survival Motor Neuron, The Gene Underlying Spinal Muscular Atrophy. Sen A, Dimlich DN, Guruharsha KG, Kankel MW, Hori K, Yokokura T, Brachat S, Richardson D, Loureiro J, Sivasankaran R, Curtis D, Davidow LS, Rubin LL, Hart AC, Van Vactor D, Artavanis-Tsakonas S.  Proceedings of the National Academy of Science.

The Dcps Inhibitor RG3039 Improves Survival, Function And Motor Unit Pathologies In Two SMA Mouse Models. Gogliotti RG, Cardona H, Singh J, Bail S, Emery C, Kuntz N, Jorgensen M, Durens M, Xia B, Barlow C, Heier CR, Plasterer HL, Jacques V, Kiledjian M, Jarecki J, Rusche J, Didonato CJ. Human Molecular Genetics.

The Dcps Inhibitor RG3039 Improves Motor Function In SMA Mice.  Van Meerbeke JP, Gibbs RM, Plasterer HL, Miao W, Feng Z, Lin MY, Rucki AA, Wee CD, Xia B, Sharma S, Jacques V, Li DK, Pellizzoni L, Rusche JR, Ko CP, Sumner CJ.   Human Molecular Genetics.

SMA Valiant Trial: A Prospective, Double-Blind, Placebo-Controlled Trial Of Valproic Acid In Ambulatory Adults With Spinal Muscular Atrophy.  Kissel JT, Elsheikh B, King WM, Freimer M, Scott CB, Kolb SJ, Reyna SP, Crawford TO, Simard LR, Krosschell KJ, Acsadi G, Schroth MK, D’Anjou G, Lasalle B, Prior TW, Sorenson S, Maczulski JA, Swoboda KJ; for the Project Cure Spinal Muscular Atrophy Investigators Network.  Muscle and Nerve.

How Genetic Modifiers Influence The Phenotype Of Spinal Muscular Atrophy And Suggest Future Therapeutic Approaches. Wirth B, Garbes L, Riessland M.  Current Opinion in Genetics and Development.

Reliability And Validity Of The TIMPSI For Infants With Spinal Muscular Atrophy Type I. Krosschell KJ, Maczulski JA, Scott C, King W, Hartman JT, Case LE, Viazzo-Trussell D, Wood J, Roman CA, Hecker E, Meffert M, Léveillé M, Kienitz K, Swoboda KJ; Project Cure SMA Investigators Network.  Pediatric Physical Therapy.

Vitamin D Intake Is Inadequate In Spinal Muscular Atrophy Type I Cohort: Correlations With Bone Health.  Aton J, Davis RH, Jordan KC, Scott CB, Swoboda KJ.  Journal of Child Neurology.

Genetic Correction Of Human Induced Pluripotent Stem Cells From Patients With Spinal Muscular Atrophy.  Corti S, Nizzardo M, Simone C, Falcone M, Nardini M, Ronchi D, Donadoni C, Salani S, Riboldi G, Magri F, Menozzi G, Bonaglia C, Rizzo F, Bresolin N, Comi GP.  Science Translational Medicine.

Nuclear Fragile X Mental Retardation Protein Is Localized To Cajal Bodies. Dury AY, El Fatimy R, Tremblay S, Rose TM, Côté J, De Koninck P, Khandjian EW.  PLoS Genetics.

SMN Is Essential for the Biogenesis of U7 Small Nuclear Ribonucleoprotein and 3′-End Formation of Histone mRNAs. Tisdale S, Lotti F, Saieva L, Van Meerbeke JP, Crawford TO, Sumner CJ, Mentis GZ, Pellizzoni L.  Cell Reports.

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