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Understanding Exon Skipping | Let's Skip Ahead

Keep me signed in. Exons, Genes, and Proteins. To better understand exon skipping, start with learning the connection between genes and proteins. Explore how exon skipping works and may help boys with Duchenne. A simple-to-use tool helps you understand the link between exon deletions and exon skips. What Is Genetic Testing? A blood test can give families vital information about Duchenne. Should I Get Tested? Make an informed choice about genetic testing by speaking with a genetic counselor. Learn the lin...

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Understanding Exon Skipping | Let's Skip Ahead | skipahead.com Reviews
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Keep me signed in. Exons, Genes, and Proteins. To better understand exon skipping, start with learning the connection between genes and proteins. Explore how exon skipping works and may help boys with Duchenne. A simple-to-use tool helps you understand the link between exon deletions and exon skips. What Is Genetic Testing? A blood test can give families vital information about Duchenne. Should I Get Tested? Make an informed choice about genetic testing by speaking with a genetic counselor. Learn the lin...
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2 new here
3 join us
4 share this
5 exon skipping
6 exon skipping explained
7 exon mapping tool
8 genetic testing
9 about duchenne
10 duchenne defined
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create account/password,new here,join us,share this,exon skipping,exon skipping explained,exon mapping tool,genetic testing,about duchenne,duchenne defined,duchenne dictionary,resources,why join us,let's skip ahead,clinical trial basics,sarepta,duchenne
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Understanding Exon Skipping | Let's Skip Ahead | skipahead.com Reviews

https://skipahead.com

Keep me signed in. Exons, Genes, and Proteins. To better understand exon skipping, start with learning the connection between genes and proteins. Explore how exon skipping works and may help boys with Duchenne. A simple-to-use tool helps you understand the link between exon deletions and exon skips. What Is Genetic Testing? A blood test can give families vital information about Duchenne. Should I Get Tested? Make an informed choice about genetic testing by speaking with a genetic counselor. Learn the lin...

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Considering genetic testing | Let's Skip Ahead

http://www.skipahead.com/should-i-get-tested

Keep me signed in. Exons, Genes, and Proteins. To better understand exon skipping, start with learning the connection between genes and proteins. Explore how exon skipping works and may help boys with Duchenne. A simple-to-use tool helps you understand the link between exon deletions and exon skips. What Is Genetic Testing? A blood test can give families vital information about Duchenne. Should I Get Tested? Make an informed choice about genetic testing by speaking with a genetic counselor. There are man...

2

Together, we can do more for Duchenne | Let's Skip Ahead

http://www.skipahead.com/why-join-us

Keep me signed in. Exons, Genes, and Proteins. To better understand exon skipping, start with learning the connection between genes and proteins. Explore how exon skipping works and may help boys with Duchenne. A simple-to-use tool helps you understand the link between exon deletions and exon skips. What Is Genetic Testing? A blood test can give families vital information about Duchenne. Should I Get Tested? Make an informed choice about genetic testing by speaking with a genetic counselor. Sarepta Thera...

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Exons, genes, and proteins | Let's Skip Ahead

http://www.skipahead.com/exon-skipping

Keep me signed in. Exons, Genes, and Proteins. To better understand exon skipping, start with learning the connection between genes and proteins. Explore how exon skipping works and may help boys with Duchenne. A simple-to-use tool helps you understand the link between exon deletions and exon skips. What Is Genetic Testing? A blood test can give families vital information about Duchenne. Should I Get Tested? Make an informed choice about genetic testing by speaking with a genetic counselor. Duchenne musc...

4

Sarepta Clinical Trials | Let's Skip Ahead

http://www.skipahead.com/sarepta-trials

Keep me signed in. Exons, Genes, and Proteins. To better understand exon skipping, start with learning the connection between genes and proteins. Explore how exon skipping works and may help boys with Duchenne. A simple-to-use tool helps you understand the link between exon deletions and exon skips. What Is Genetic Testing? A blood test can give families vital information about Duchenne. Should I Get Tested? Make an informed choice about genetic testing by speaking with a genetic counselor. If you are in...

5

Clinical trials | Let's Skip Ahead

http://www.skipahead.com/clinical-trials

Keep me signed in. Exons, Genes, and Proteins. To better understand exon skipping, start with learning the connection between genes and proteins. Explore how exon skipping works and may help boys with Duchenne. A simple-to-use tool helps you understand the link between exon deletions and exon skips. What Is Genetic Testing? A blood test can give families vital information about Duchenne. Should I Get Tested? Make an informed choice about genetic testing by speaking with a genetic counselor. Before any dr...

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sarepta.com sarepta.com

Locations | Sarepta Therapeutics

http://www.sarepta.com/company/locations

Skip to main content. Cambridge, MA 02142. Main phone: 617.274.4000. General email: info@sarepta.com. 4575 SW Research Way, Suite 200. Corvallis, OR 97333. A resource center for families living with DMD.

sarepta.com sarepta.com

Career Opportunities | Sarepta Therapeutics

http://www.sarepta.com/our-team/career-opportunities

Skip to main content. To apply for an open position, please select a job title below, complete the online application form, and upload your resume and cover letter. You may also choose to create a profile if you wish to apply for other opportunities. To send a resume via an alternative method, please mail it to our corporate address: Sarepta Therapeutics, Attention: Human Resources, 215 First Street, Cambridge, MA 02142. A resource center for families living with DMD.

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Our Pipeline | Sarepta Therapeutics

http://www.sarepta.com/our-pipeline

Skip to main content. Our rapidly advancing clinical programs in Duchenne muscular dystrophy, Marburg and influenza represent the foundation for a growing and diverse pipeline in rare and infectious diseases. Our therapeutic development programs include lead clinical candidates for Duchenne muscular dystrophy and infections of public health significance, including influenza and multi-drug resistant bacterial infections. DMD EXON 51 (ETEPLIRSEN / AVI-4658). DMD EXON 53 (SRP-4053). DMD EXON 45 (SRP-4045).

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Corporate Overview | Sarepta Therapeutics

http://www.sarepta.com/our-company

Skip to main content. Our vision is to transform how the world approaches the treatment of serious and life-threatening diseases by unlocking the potential of RNA-based technologies. Recognizing the complexity of this goal, we focus on rare and infectious diseases with tremendous unmet needs areas in which new therapies have the potential to make dramatic improvements in the lives of patients. A resource center for families living with DMD.

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Disease Resources | Sarepta Therapeutics

http://www.sarepta.com/community/disease-resources

Skip to main content. Duchenne muscular dystrophy (DMD) is a rare, fatal neuromuscular genetic disease that occurs in approximately one in every 3,500 5,000 males worldwide. DMD is caused by a change or mutation in the gene that encodes instructions for dystrophin. These changes in the genetic code may include large deletions (about 60-70 percent), large duplications (about 10 percent) or other small changes (about 15-30 percent). The role of dystrophin. Symptoms of DMD usually appear in infants and todd...

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AVI-7288 for Treatment of Marburg Virus | Sarepta Therapeutics

http://www.sarepta.com/pipeline/avi-7288-marburg

Skip to main content. We are developing AVI-7288 for the treatment of Marburg virus infection under a contract with the U.S. Department of Defense managed by the Medical Countermeasure Systems BioDefense Therapeutics (MCS-BDTX) Joint Product Management Office. AVI-7288 is based on our proprietary next-generation PMO plus. Chemistry, which is also used across our infectious disease pipeline, including our lead investigational candidate for influenza. Clinical development of AVI-7288 is being conducted pur...

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Board of Directors | Sarepta Therapeutics

http://www.sarepta.com/company/board-directors

Skip to main content. M KATHLEEN BEHRENS, Ph.D. RICHARD J. BARRY. JEAN-PAUL KRESS, M.D. CLAUDE NICAISE, M.D. HANS WIGZELL, M.D., Ph.D. M KATHLEEN BEHRENS, P h. M Kathleen Behrens, Ph.D., has served as a member of our Board since March 2009 and Chairwoman of the Board since April 2015. She also currently serves as a member and chair of the audit committee and as a member of the research and development committee of the Board. Richard J. Barry. Mr Barry holds a B.A. from Pennsylvania State Universi...Dr Kr...

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Exon-Skipping for Duchenne | Sarepta Therapeutics

http://www.sarepta.com/pipeline/exon-skipping-duchenne

Skip to main content. DMD EXON 51 (ETEPLIRSEN / AVI-4658). DMD EXON 53 (SRP-4053). DMD EXON 45 (SRP-4045). DMD EXON 52 (SRP-4052). The underlying cause of DMD is a mutation or error in the gene for dystrophin, an essential protein involved in muscle fiber function. Our investigational therapies for DMD are designed to skip an exon in the dystrophin pre-m RNA to enable the synthesis of a functional shorter form of the dystrophin protein. EXPLORE OUR TECHNOLOGY FOR DMD. 2009 Mar;30 (3):293-9.

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Understanding Exon Skipping | Let's Skip Ahead

Keep me signed in. Exons, Genes, and Proteins. To better understand exon skipping, start with learning the connection between genes and proteins. Explore how exon skipping works and may help boys with Duchenne. A simple-to-use tool helps you understand the link between exon deletions and exon skips. What Is Genetic Testing? A blood test can give families vital information about Duchenne. Should I Get Tested? Make an informed choice about genetic testing by speaking with a genetic counselor. Learn the lin...

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