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…
According to a story from BioPharma Dive, the US Food and Drug Administration (FDA) has recently approved Beqvez, a gene therapy from pharmaceutical giant Pfizer intended to treat hemophilia B.…
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…
Duchenne muscular dystrophy (DMD) is an inherited disease that weakens a child’s muscles and eventually spreads throughout the child’s body. DMD is caused by mutations in the gene that helps…
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…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
