Imagine a large pickle jar, the kind you might see at a movie concession stand filled with pickles. But inside this otherwise empty jar sits a large ball that’s pressing against the sides and all but filling it. Then imagine that this ball symbolizes grief. Many believe that…
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Researchers from the Republic of Korea developed a new type of needle that is able to completely prevent bleeding following syringe needle puncture. This new type of needle can be invaluable for people with blood clotting disorders like hemophilia where syringe injections can have significant side effects such as uncontrolled bleeding.
An experimental form of genome editing called clustered regularly interspaced short palindromic repeats or CRISPR/Cas9 could offer a faster and cheaper method of treating people with hemophilia, compared to the current DNA editing method. Because hemophilia is due to DNA mutations, the genome editing could be used to efficiently slice at the DNA,…
My mighty warrior Caeleb came home from school last week with a question. “Mom,” he asked, “my knee is really messed up, isn’t it?” I couldn’t see an injury, and so I asked him what he meant by “messed up.” “I can’t run really fast like everyone else in gym class,”…
Hemgenix (etranacogene dezaparvovec), formerly known as AMT-061 and EtranaDez, is a gene therapy given as a one-time intravenous (into-the-vein) infusion to treat bleeding episodes in hemophilia B.
“Be prepared.” Scouts around the world have this motto ingrained in their hearts. While I was a Girl Scout for only a short time, this practice remains essential to me. I look ahead and consider the numerous ways a situation may manifest. As the mother of two sons with…
Data from a completed Phase 2b trial indicate that dalcinonacog alfa (DalcA) is an effective and safe therapy to prevent bleeding in people with severe hemophilia B. Final results from this open-label study in six patients are expected by June. “We are pleased to have successfully completed the DalcA Phase…
Every family member has a role. Parents usually assume the roles of providers and nurturers, making sure their children have a roof over their heads, food to eat, and plenty of love. Siblings each bring their unique personalities to the home. Some are playful and fun-loving, while others may be…