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NIH Conference and Legislative Update
…Workshop, please go to http://www.sonicfoundry.com/NIH. With regard to the SMA Treatment Acceleration Act, despite our best efforts and the unbelievable show of support by families across the nation, the current…
Read MoreBiogen May Spend as Much as $299 Million on Isis Treatment for Children
…both based in Basel, Switzerland, are also testing drugs for the disease. “I am very, very excited about this compound,” said Alfred Sandrock, a senior vice president at Biogen, in…
Read MoreDrug Development Process
…The goal of lead optimization is to identify a compound (Development Candidate) suitable for preclinical development. Preclinical Development: This stage consists of testing a Development Candidate in various in vitro…
Read MoreDrug Discovery
…vitro systems to screen existing drugs and compound libraries. Over 120 such compounds were screened in mouse models of severe SMA, but none produced more than a modest effect on…
Read MoreSMA Journal Articles
Research: SMA Journal Articles Research: SMA Journal Articles A selection of recent articles in the field of SMA is provided below. For a complete listing of SMA research articles, please…
Read MoreDrug Provides Glimmer of Hope for Children Suffering Fatal Genetic Disease
…SMA. He’s currently conducting a clinical trial to test whether the treatment can slow or halt the progression of the disease in affected children. The genetics of SMA are complicated….
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