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SMN2
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#	Item
1	Dear Spinal Muscular Atrophy Community Members, We would like to update you on the clinical development of RG7800, an investigational SMN2 splicing modifier that is being studied as a potential treatment for spinal muscu Add to Reading List Source URL: www.sma-schweiz.ch Language: English - Date: 2015-05-07 03:16:04 Motor neurone disease Spinal muscular atrophy
2	npg Call in the backup The most common genetic killer of infants, a disease known as spinal muscular atrophy, is caused by mutations in a single gene. The human genome contains its own backup system—near-identical cop Add to Reading List Source URL: www.fsma.org.hk Language: English - Date: 2013-04-29 02:31:53 Motor neurone disease Spinal muscular atrophy RNA splicing Applied genetics Biotechnology SMN1 Antisense therapy Survival of motor neuron SMN2 Genetics Biology Spliceosome
3	ACOG COMMITTEE OPINION Number 432 • May 2009 Spinal Muscular Atrophy Committee on Genetics Add to Reading List Source URL: www.acog.org Language: English - Date: 2014-12-22 17:23:44 Medical genetics Rare diseases SMN1 Spinal muscular atrophies Survival of motor neuron Tay–Sachs disease SMN2 Genetic counseling Genetic testing Medicine Health Motor neurone disease
4	Hindawi Publishing Corporation Journal of Neurodegenerative Diseases Volume 2013, Article ID[removed], 7 pages http://dx.doi.org[removed][removed]Clinical Study Add to Reading List Source URL: downloads.hindawi.com Language: English - Date: 2014-03-26 09:30:43 Medical genetics SMN1 Spinal muscular atrophies SMN2 Survival of motor neuron Chromosome 5 Muscle atrophy Hypotonia Amyotrophic lateral sclerosis Motor neurone disease Health Medicine
5	Spinal Muscular Atrophy & ISIS-SMNRx Isis Pharmaceuticals is developing a drug to treat Spinal Muscular Atrophy (SMA). SMA is a genetic neuromuscular disease characterized by muscle atrophy and weakness. SMA is a leading Add to Reading List Source URL: www.muscle.ca Language: English - Date: 2013-11-30 18:15:27 Genetic genealogy Spinal muscular atrophy SMN1 Survival of motor neuron Antisense therapy Amyotrophic lateral sclerosis SMN2 Sense Gideon Dreyfuss Motor neurone disease Biology Genetics
6	SMA CARE SERIES The Genetics of Spinal Muscular Atrophy SMA CARE SERIES - The Genetics of Spinal Muscular Atrophy Add to Reading List Source URL: www.curesma.org Language: English - Date: 2014-08-20 18:41:49 Motor neurone disease RNA splicing Spliceosome DNA SMN1 SMN2 Chromosome 5 Survival of motor neuron Gene Biology Genetics Philosophy of biology
7	# Phase I Gene Therapy Trial for SMA Delivering the Survival Motor Neuron Gene by scAA9 RAC Protocol[removed] Add to Reading List Source URL: osp.od.nih.gov Language: English - Date: 2014-01-13 12:36:03 Health Spinal muscular atrophy SMN1 Survival of motor neuron SMN2 Amyotrophic lateral sclerosis Hypotonia LSm Neuron Motor neurone disease Genetic genealogy Medical genetics
8	A Tetracycline That Corrects SMN2 Splicing for Potential Treatment of Spinal Muscular Atrophy (SMA) Poster # [removed]Kneeland Street Add to Reading List Source URL: www.paratekpharm.com Language: English - Date: 2012-10-12 16:03:38 Gene expression RNA splicing Motor neurone disease Spinal muscular atrophy RNA SMN1 SMN2 Survival of motor neuron Alternative splicing Biology Genetics Spliceosome
9	Spinal Muscular Atrophy In Focus Dear Readers Fast Facts Add to Reading List Source URL: mdausa.org Language: English - Date: 2012-06-12 18:41:13 Spliceosome RNA splicing Spinal muscular atrophy RNA DNA SMN1 Survival of motor neuron SMN2 LSm Genetics Biology Motor neurone disease
10	Families of Spinal Muscular Atrophy Transcript of Chat Live with an Expert December 1, 2004 Topic: Genetics and SMA Our expert is Louise Simard, PhD, and SMA researcher at Montreal’s Saint Justine Hospital. Add to Reading List Source URL: www.fsma.org Language: English - Date: 2008-02-28 15:54:00 Emerging technologies Stem cells Cell biology SMN1 Neuron Stem cell treatments SMN2 Gene therapy Amyotrophic lateral sclerosis Biology Motor neurone disease Biotechnology

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