5 Things to Know about Spinal Muscular Atrophy скачать в хорошем качестве

5 Things to Know about Spinal Muscular Atrophy

Two years ago, St. Jude Children’s Research Hospital expanded its mission to research, treat and cure childhood neurological disorders through the Pediatric Translational Neuroscience Initiative (PTNI), a program designed to propel effective and innovative treatments from the lab to the clinic. Richard Finkel, MD, joined St. Jude to lead the clinical component of the program. Before coming to St. Jude, Finkel played a vital role in testing what eventually became three successful drugs for SMA, a genetic disorder characterized by muscle weakness and wasting. Finkel has launched several clinical trials at St. Jude to treat SMA and other childhood neurologic diseases. He shares five things to know about spinal muscular atrophy. 1. Spinal muscular atrophy used to be the most common genetic cause of infant mortality. 2. SMA can affect children and adults of all ages. And yet, there is still no cure. 3. Recent scientific discoveries have led to the development of three drugs that increase both survival and function of these children and adults with SMA. But there is still no cure, and there’s a lot of work to be done. 4. As a doctor and clinical scientist who has seen these children and adults with SMA for several years, Finkel has been able to participate in the clinical trials for the development of the three drugs that are now available to treat these patients. 5. St. Jude now has two clinical trials to push our understanding forward to try to develop new and innovative treatments. Explore open jobs: http://www.stjude.org/joinourmission Follow @StJudeResearch across social media to explore our science and clinical care. Twitter: https://bit.ly/StJude-Twitter Facebook: https://bit.ly/StJude-Facebook Instagram: https://bit.ly/StJude-Instagram LinkedIn: https://bit.ly/StJude-LinkedIn Mastodon: https://bit.ly/StJude-Mastodon #FiveThingsToKnow #Neuroscience #Neurology #SpinalMuscularAtrophy #StJude #StJudeResearch #RareDisease