March 9, 2010
A New Finding Clarifies the Cause of Spinal Muscular Atrophy

February 24, 2008
"A Disease in the Crosshairs"
Researchers select SMA as a match for their patented gene therapy. - Read more...

September 29, 2007
A biopharmaceutical company in France announces it is enrolling patients in a clinical trial for SMA type I.
"Trophos Starts Phase Ib Clinical Trial of TRO19622 In Spinal Muscular Atrophy" - Read more...

Feb.22, 2007
"Treatment Extends Survival in Mouse Model of Spinal Muscular Atrophy" - Read more...


Research Publications

Combination of SMN2 copy number and NAIP deletion predicts disease severity in spinal muscular atrophy. Watihayati MS, Fatemeh H, Marini M, Atif AB, Zahiruddin WM, Sasongko TH, Tang TH, Zabidi-Hussin ZA, Nishio H, Zilfalil BA. Brain Dev. 2009 Jan;31(1):42-5.

Induced pluripotent stem cells from a spinal muscular atrophy patient. Ebert AD, Yu J, Rose FF Jr, Mattis VB, Lorson CL, Thomson JA, Svendsen CN. Nature. 2008 Dec 21.

Multiple therapeutic effects of valproic acid in spinal muscular atrophy model mice. Tsai LK, Tsai MS, Ting CH, Li H.J Mol Med. 2008 Nov;86(11):1243-54.

Sustained improvement of spinal muscular atrophy mice treated with trichostatin A plus nutrition. Narver HL, Kong L, Burnett BG, Choe DW, Bosch-Marcé M, Taye AA, Eckhaus MA, Sumner CJ. Ann Neurol. 2008 Oct;64(4):465-70.

Pathogenesis of proximal autosomal recessive spinal muscular atrophy. Simic G. Acta Neuropathol. 2008 Sep;116(3):223-34.

A natural history study of late onset spinal muscular atrophy types 3b and 4. Piepers S, van den Berg LH, Brugman F, Scheffer H, Ruiterkamp-Versteeg M, van Engelen BG, Faber CG, de Visser M, van der Pol WL, Wokke JH. J Neurol. 2008 Sep;255(9):1400-4.

Unaffected patients with a homozygous absence of the SMN1 gene. Jedrzejowska M, Borkowska J, Zimowski J, Kostera-Pruszczyk A, Milewski M, Jurek M, Sielska D, Kostyk E, Nyka W, Zaremba J, Hausmanowa-Petrusewicz I. Eur J Hum Genet. 2008 Aug;16(8):930-4.

Genetic conversion of an SMN2 gene to SMN1: a novel approach to the treatment of spinal muscular atrophy. DiMatteo D, Callahan S, Kmiec EB. Exp Cell Res. 2008 Feb 15;314(4):878-86

Valproic acid treatment in six patients with spinal muscular atrophy. Tsai LK, Yang CC, Hwu WL, Li H. Eur J Neurol. 2007 Dec;14(12):e8-9.

Differential SMN2 Expression Associated with SMA Severity. Gavrilov DK, Shi X, Das K, Gilliam TC, Wang CH. Nature Genetics. 1998;20: 230-231.

For more information about SMA laboratory and clinical research, visit the PubMed Database.