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Two gene therapies that the Food and Drug Administration describes as milestones were approved Friday for treatment of sickle cell disease.The Centers for Disease Control and Prevention says 100,000 Americans are living with sickle cell disease, which disproportionately affects Black and Hispanic individuals. The primary problem in sickle cell disease is a mutation in hemoglobin, a protein found in red blood cells that delivers oxygen to the bodys tissues, the FDA states.The mutation causes red blood cells to change shape, which restricts the flow of blood to vessels, causing severe pain and organ damage.The two stanley us new therapies edit a person s genes to make sure the body produces blood cells tha stanley taza t an adult without the disease would produce, the FDA says. Both therapies have been approved for people 12 years and older. Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited, said Dr. Nicole Verdun, director of the FDA s Office of Therapeutic Products.SEE MORE: 2 babies infected with rare bacteria found in powdered infant formulaPeople who receive either of the two new treatments will be followed for a long-term study to evaluate the product s safety and effectiveness, the FDA said. Todays actions follow rigorous evaluations of the scientific and clinical data needed to support approval, reflecting the FDAs commitment to stanley cup facilitating development of safe an Zkgk MCTS reveals new bus shelters along S. 27th St.
Focus on peoples capabilities, not their disabilities. Thats the message behind the Dan Marino Foundation. Its difficult for our children with autism. A lot of people, they see them, maybe theyre doing different cups stanley things, and people dont alwa caneca stanley ys accept that, said Susan Morantes.Morantes has a son on the autism spectrum. Susan said Sean had become the man he is today because of the Dan Marino Foundation. Sean was diagnosed with autism at four back when a lot of people didnt know about autism, so it was very difficult to find resources at that point in time. By luck, my husband stanley cup nz was able to meet Dan, and ever since, Dan and Claire have been mentors to us, she said.The Dan Marino Foundation empowers and reduces the over 80% unemployment rate for individuals with autism and other developmental disabilities. The foundation focuses on creating opportunities leading to employment and independence. At the beginning, thats what he was learning, just how to navigate employment scenarios. My son probably has the most challenges in social activities, so just to be careful not to say the wrong thing to people, not to do the wrong thing, how to talk to people, how to engage people, said Morantes.The vocational rehabilitation career camp, offered to students ages 14 to 21, is a week-long program that assists with resume writing, job search, career goal development, and leadership skills. Were focusing on self-advocacy, so that could look like learning about the Americans Disabilities Act and |
