β-thalassemia major is a rare blood condition that necessitates a lifetime of blood transfusions. Despite advancements in treatments, the perfect cure has yet to be found. Bluebird Bio, Inc. is…
Have you ever heard of “ex vivo” gene therapy? If you haven't, it means removing some cells, modifying them, and putting them back in. The goal? Curing the diseases that…
Medicine is expensive. Most patients aren’t millionaires. It’s disheartening that treatments which could be so beneficial are out of reach for so many. It’s even more disheartening when their production…
La terapia genética se ha hablado desde hace bastante tiempo, pero las noticias recientes han convertido la esperanza en realidad para los bebés nacidos con adenosina desaminasa (ADA). Los bebés…
It’s always heartening to see scientists take up research in therapeutic areas that are under served and poorly understood. And when their research yields positive results, it’s downright amazing. Honestly,…
Podría ser una historia de una novela de espías: Si bien se están pasando mensajes codificados, un intruso intenta infiltrarse e interrumpir la comunicación. Parte de la misión implica el…
It takes an average of seven years for a patient with a rare disease like severe combined immunodeficiency (SCID) to receive a correct diagnosis; and that comes after the patient…
Gene therapy is the hot new thing in medicine these days--especially with hemophilia. The good new is, “Yay! There are hot new things in medicine exciting researchers!” The bad news…
A cure may be just around the corner for people who have hemophilia B. And anytime the word "cure" appears in a sentence with a disease state, there's cause for…
Hunter Syndrome, is a heartbreaking twist of genetic fate, and we celebrate that the FDA has granted Rare Pediatric Disease Designation to REGENXBIO's RGX-121 Gene Therapy. Hunter Syndrome causes seemingly…
While it wouldn’t be fair to describe hemophilia B as the red-headed stepchild of the bleeding disorder community, it is far rarer than the more common hemophilia A, and bleeds…
One of the many problems with having a rare disease is that very few people know anything about what you, or your loved one, go through on a day-to-day basis.…
You may have heard the buzz words "gene therapy" without really understanding what it is, or what's at stake. Gene therapy is when a healthy copy of a gene replaces…
Podría ser una historia de una novela de espías: Si bien se están pasando mensajes codificados, un extraño intenta infiltrarse e interrumpir la comunicación. Parte de la misión involucra el…
At PatientWorthy, we've talked a lot about gene therapy, but we've never really taken the time to explain it. What the heck is gene therapy? How does it work? Well, like…
It could be a story out of a spy novel: While coded messages are being passed, an outsider attempts to infiltrate and disrupt communication. Part of the mission involves smuggling important equipment…
Gene therapy has been talked about for quite some time, but recent news has turned a hope into a reality for babies born with adenosine deaminase (ADA)-deficient SCID, severe combined…
For many people suffering from rare diseases, gene therapy offers the hope for relief and perhaps a cure for their particular condition. But while many studies are underway, to date, few drugs have…
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