People with spinal muscular atrophy and other rare conditions with either few or no approved treatments have recently had their hope of a cure renewed by the concept of gene therapy. Basically,…
Last week the Biotechnology Innovation Organization released a letter to the FDA, commenting on the latter's draft of a document that proposes a new set of guidelines for the treatment…
Two children from a Brooklyn family were born with an ultra rare genetic disease called Canavan disease. Their parents' research revealed that gene therapy could possibly treat the disease. Unfortunately,…
A gene therapy treatment is being used to treat X-linked severe combined immunodeficiency (X-SCID). Mustang Bio has licensed the treatment from St. Jude Children's Research Hospital. X-SCID is also known…
In a Washington Post article by Ben Guarino, new cancer treatments lasso the power of naturally occurring immune cells in the body. Researchers are looking into ways modify them to…
Gene therapy is on the rise, and it just might be exactly what many children suffering from genetic disorders need to save their lives. Today reported that an amazing organization…
Minoryx Therapeutics is on their way to making strides for patients with adrenomyeloneuropathy, also known as AMN, reports Labiotech. They just started their phase II initiative to test patients for…
At six years old, Benny was unable to sit up, roll over and scoot like other babies his age. Missing all the major milestones, his mother Jennie knew that something…
A new gene therapy treatment may be on the horizon for Oculopharyngeal Muscular Dystrophy patients. Find out more below, or keep up with the original story at musculardystrophynews.com. Oculopharyngeal Muscular…
Earlier this month, PBS released the "The Gene Doctors." The hour-long documentary follows the recent emergence of gene therapy as a novel method of treating rare diseases. The film discusses…
As the world turns, so do advancements in gene therapy. Technology is changing our world so drastically that soon gene therapy will be simplified into a one-shot treatment. With it,…
Sickle cell anemia results from a single mutation in the hemoglobin gene. The mutation creates a protein which has an atypical structure. This altered structure throws a wrench in the…
In the ultimate feat of recycling, scientists at UCLA and the Howard Hughes Medical Institute have re-purposed the LeXis gene as a cholesterol-lowering agent. A recent press release issued by…
Little Emily Whitehead is looking attentively at a butterfly that has landed on her finger. She coos at it and lets it rest on the tip of her thumb peacefully.…
Pharma giant Novartis's new cancer drug tisagenlecleucel passed a critical test by winning the support of a federal advisory panel - setting it up for final FDA approval in September.…
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…
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