As reported on Drugs.com, REGENXBIO has announced that the U.S. Food and Drug Administration (FDA) has halted clinical testing of its investigational gene therapies RGX‑111 and RGX‑121, two programs targeting…
As discussed in a recent interview between Krystal Jacques and Prof. Brian Bigger, a novel stem cell–based gene therapy is showing early promise as a potential one-time, lifelong treatment for…
As reported on MedicalXpress, esearchers at the Children’s Hospital of Philadelphia (CHOP) and the University of Pennsylvania are pioneering a new approach to treating rare genetic disorders using CRISPR-based therapies.…
Please go HERE to read Part 1 of this article The company, GEMMABIO, will pursue a number of rare, large indications. A new spinout, RareTx, will be dedicated to Orphan…
As noted in a recent article published in Inside Precision Medicine, in 1992 gene therapy was often discussed but seldom practiced. When Jim Wilson MD, PhD attended Penn State, gene…
Lexeo Therapeutics announced a significant regulatory development this week: the U.S. Food and Drug Administration (FDA) is open to a faster approval pathway for the company’s promising gene therapy, LX2006,…
Editor's Note: Patient Worthy is proud to share this article from our friends at the TESS Research Foundation. To see the article in its original format, please click here. TL;DR:…
Gene therapy for hemophilia B has reached a significant milestone: more than a decade after a single treatment, patients continue to experience stable benefits with minimal safety concerns, according to…
A groundbreaking study has demonstrated that gene therapy can successfully restore hearing in patients with hereditary deafness, offering new hope for individuals affected by genetic forms of hearing loss. The…
The treatment landscape for hemophilia B has taken a historic leap forward, as the first patient in the UK’s National Health Service (NHS) has received Hemgenix, a pioneering gene therapy…
As reported by HD Buzz, a significant step forward for Huntington’s disease (HD) research, Roche has announced that the first patient has been dosed in its new gene therapy clinical…
Researchers have discovered a potential “window” whereby babies born with inherited diseases may be treated with gene therapy sent directly into their circulatory systems. The window consists of circulating stem…
Many people with rare diseases struggle to access personalized therapies simply because they lack a confirmed genetic diagnosis. This barrier blocks access to targeted treatments and clinical trials, leaving families…
In a groundbreaking medical achievement, physicians have administered the world’s first personalized gene-editing therapy to an infant suffering from a rare and fatal genetic disorder. The innovative treatment, described at…
An article titled “Accelerated Approval as the New Norm in Gene Therapy for Rare Diseases” from DIA Global Forum digital magazine’s May 2025 issue explores the evolving landscape of regulatory…
In 2023, Marley Mansour of Rowlett, Texas and her family were told that Marley had an extremely rare genetic condition called NARS1 disorder. Now, Marley is the world’s first person…
The use of the gene therapy elivaldogene autotemcel (Skysona), has been approved due to its success in the treatment of patients diagnosed with the neurological disease cerebral adrenoleukodystrophy (CALD). The…
Researchers discovered a version of the Cas protein that fits into adeno-associated viruses (AAVs). This virus can be engineered to deliver targeted therapy to cells and is a common approach…
Yusara Ahmed was prepared to take action when she learned that her son Yusuf was diagnosed with beta thalassemia, an uncommon blood disorder. Yusara's sister also lived with the disease,…
Recently there has been an increasing amount of research attempting to control CRISPR gene-editing systems. These systems are found in many types of bacteria as a defense against viruses. The…
Pfizer has recently paused its Phase III clinical trial that has been assessing the Duchenne muscular dystrophy candidate fordadistrogene movaparvovec. The Phase II DAYLIGHT trial (NCT05429372), enrolled ten boys ages…
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science…
You might have heard of Orphan Drug designation - and maybe even Rare Pediatric Disease designation. But have you heard of Regenerative Medicine Advanced Therapy (RMAT) designation? Described in Section…
The American Society of Gene and Cell Therapy (ASGCT) held its 27th Annual Meeting from May 7-11, 2024. During the conference, stakeholders in the field of cell therapy come together…
For people with geographic atrophy, also known as advanced dry age-related macular degeneration, slowing vision loss is at the top of the list in terms of goals. Treatments like SYOFOVRE…
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