Nusinersen is safe and well-tolerated, the study reports. Although the multisite trial included only 20 children and was intended primarily as a safety test, the investigators report significant improvements in patients’ ability to achieve motor milestones, as well as better motor function and increased function of nerves that are attacked by the disease. The drug is quickly progressing through the regulatory approval process. In addition to the phase-2 trial in which Zoe participated, nusinersen has been evaluated in a phase-3 trial of SMA-1 patients, which was stopped early in August because it was obvious that infants receiving the drug were achieving significantly more motor milestones than those in the control group. The phase-3 trial is now an open-label study, meaning that all participants can receive nusinersen. The drug is expected to receive approval from the FDA within the next two months, and in the interim is available under an expanded access program at a few sites around the country, including Lucile Packard Children’s Hospital Stanford. “This drug really, really, completely turns things around for SMA,” Day said. “It’s huge.” An even larger discovery is that drugs with the same mechanism of action may help treat other genetic diseases, he added. http://foresthillmotel.com/matthewowenspro/2016/11/08/considering-core-issues-in-doctorNusinersen is an antisense oligonucleotide, which works by sticking to a specific piece of genetic material. Trials of antisense oligonucleotide drugs are now underway for other neurological diseases, including muscular dystrophy, Huntington’s disease and amyotrophic lateral sclerosis.
For the original version including any supplementary images or video, visit https://www.eurekalert.org/pub_releases/2016-12/sumc-spi120516.php
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