Vision loss can be challenging. If you could have a one-time gene therapy that would improve your visual acuity, would you take it? It seems like a no-brainer. That's what…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
A study by a team of scientists at the Children’s Research Institute was reported this week in the journal Nature Communications. The study is welcomed by the medical profession as…
A majority of available treatment options for metachromatic leukodystrophy (MLD) rely on relieving symptoms. However, the recent approval of Lenmeldy (atidarsagene autotemcel) is the first ever FDA-approved gene therapy option…
Children with late-infantile onset Batten disease (CLN2) may be treated with an enzyme replacement therapy (ERT). However, this therapy cannot reverse the damage or progression children have already experienced. Researchers…
According to a story from BioSpace, the current standard approaches for drug development aren't well suited for rare diseases, which have unique characteristics in comparison to more widespread illnesses. As…
According to information in a recent press release from Wave Life Sciences, dosing has begun in a clinical trial investigating the RNA editing candidate, WVE-006. The drug treats alpha-1…
A Baylor College team of researchers headed by author and Associate Professor of Pathology, Liaising Yen, have spent over ten years working on a technology to effectively regulate gene expression.…
The Role of AI in Predicting Stem Cell Donor Availability Webinar January 31, 2024 The success of unrelated donor stem cell transplants depends on not only finding genetically matched donors…
The agency in Guangzhou, China, that arranged three-year-old Shelby’s adoption by a family in the US disclosed to Michelle and Adam Campbell that Shelby had beta thalassemia disorder. Yet, that…
Genes hold the key to our health. Through revolutionary gene therapy solutions, the medical field is transforming to laud the power and precision of personalized medicine. Gene therapy has the…
A recent release published in BioSpace announced that a gene therapy company is vigorously pursuing the study of AVB-101 to treat patients with frontotemporal dementia (FTD), including FTD with GRN…
Vascular endothelial growth factor (VEGF) has been proven to increase how permeable blood vessels are in the eyes, causing them to leak. When new blood vessels form, these may also…
If Wave Life Sciences’ recently submitted trial application for WVE-006 is approved, it will become the first RNA editing treatment in a clinical development setting. Wave’s CEO, Paul Bolno,…
For over four decades, the aspiration of gene therapy has been to develop novel therapies with the potential to enhance human health. Recombinant AAV (rAAV), a type of gene therapy,…
617 words (source - 3% match) vs. 452 words (mine - 4% match) As our healthcare field continues to innovate and grow, we have seen more conversations regarding gene therapy…
Galila Yohannes’ parents, who originally hail from Eritrea, brought their family to Israel for a better life. And while they never would have expected what came next, this move likely…
In May 2023, the U.S. Food and Drug Administration (FDA) approved Vyjuvek, a gene therapy, for dystrophic epidermolysis bullosa (DEB). This treatment has brought support to numerous people living with…
Advances in research and medicine have improved the life expectancy for people with cystic fibrosis. But this doesn't mean that we should stop searching for a cure. Most likely,…
It looked as if the fate of Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy was pretty much sealed until an FDA official intervened. This information was provided by…
For the first time ever, people living with severe hemophilia A will have access to a gene therapy solution designed to treat their condition at the source. In a…
uniQure, a leading gene therapy company, recently shared an update on a Phase 1/2 clinical study evaluating AMT-130 for Huntington’s disease. The safety and proof-of-concept study enrolled 26 participants with…
Gene therapy has the potential to improve outcomes among individuals across various disease spectrums. In the case of clinical-stage biotechnology company Rocket Pharmaceuticals, the company is working to develop…
In August 2022, I reported on a story that touched my heart: Lucy Landman's PGAP3 diagnosis. With less than 50 diagnosed cases of PGAP3 worldwide, parents Zach and Geri jumped…
According to a story from BioPharma Reporter, the company bluebird bio has recently submitted its Biologics License Application (BLA) to the US Food and Drug Administration (FDA). This application is…
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