A recent milestone in gene therapy is transforming the outlook for people with rare genetic skin disorders, signaling a new era of personalized medicine and hope for patients who previously…
For families affected by rare genetic skin diseases, hope once meant daily bandages and vigilant wound care. That began to change in 2023, when US regulators approved the first-ever gene…
A significant step forward in the treatment of a rare and painful skin condition has emerged from Finland. According to News Medical Life Sciences, Tampere University has announced the licensing…
Epidermolysis Bullosa (EB) is a devastating and rare genetic skin disorder often described as having "butterfly skin." For those living with EB, even the gentlest touch can lead to painful…
In North Texas, a remarkable young girl named Isabella "Izzy" Esquivel has found a unique way to experience happiness and self-expression—through her art. Izzy, now ten years old, was diagnosed…
Clinical-stage biotechnology company Abeona Therapeutics ("Abeona") is working to develop a novel cell therapy for people living with recessive dystrophic epidermolysis bullosa (RDEB), a rare genetic skin disorder characterized by…
There are a number of symptoms and characteristics associated with Kindler epidermolysis bullosa (Kindler EB), a rare genetic disorder. When researchers wanted to understand whether hypoplastic pitted amelogenesis imperfecta (AI),…
Healio Psoriatic Disease recently reported that Vyjuvek (beremagene geperpavec-svdt), a topical gene therapy designed for people with dystrophic epidermolysis bullosa (DEB), was granted Orphan Drug designation (ODD) by the Japanese…
In March 2022, the U.S. Food and Drug Administration (FDA) decided against the approval of oleogel-S10 for epidermolysis bullosa (EB); the FDA noted that, prior to approval, it would need…
In 2010, Eddie Vedder (well known for his musical career in Pearl Jam) co-founded the EB Research Partnership (EBRP) alongside wife Jill and a group of parents whose children…
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…
Managing dystrophic epidermolysis bullosa (DEB) can be difficult. DEB is a rare genetic condition which causes fragile, easily blistered skin. There are multiple DEB subtypes with their own inheritance…
According to a story from VICE News, Congressman George Santos was recently elected to Congress in the 2022 mid-term elections. Since then, his past has been placed under scrutiny, and…
Lucy Beall's parents were expecting a fairly normal and straightforward birth. But when Lucy was born, the entire room was shocked. She weighed just six pounds and her skin…
Before you read on, make sure to check out Part 1 of our interview, where Annie, Peter, and Patterson discuss what recessive dystrophic epidermolysis bullosa (RDEB) is, Patterson's diagnostic journey, and how…
Annie and Peter McKenzie describe their seven-year-old son Patterson as having a “heart of gold.” Patterson is bright, funny, happy, and empathetic. Despite his insistence that he is not a…
Did you know that bone mass accrual and bone mineral density depend highly on puberty? Because of this, puberty delays or interruptions can negatively affect bone mineral density or cause…
Clinical trials are greatly beneficial in understanding more about therapeutics and various conditions. For example, the Phase 3 VIITAL trial will evaluate the safety, efficacy, and tolerability of EB-101, an…
Within 48 hours following her son Kylan's birth, Emily Bussenschutt had already visited three separate hospitals searching for a diagnosis. 9Honey explains that Kylan was born with epidermolysis bullosa…
People with epidermolysis bullosa (EB) often experience symptoms such as intense itching or severe pain. Unfortunately, these symptoms can be debilitating and may greatly affect overall quality-of-life. However, some burgeoning…
Those with recessive dystrophic epidermolysis bullosa (RDEB) have fragile, easily blistered skin. Unfortunately, because of dysfunctional tissue repair, wounds may take longer to heal – causing increased levels of scarring…
CONTENT WARNING: Severe skin wounds and damage Working in rare disease is always hard- It means working in suffering and grief, but even with all our experience, we had to…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
In early June 2021, Epidermolysis Bullosa News reported that Filsuvez (Oleogel-S10), a topical gel for epidermolysis bullosa (EB), received Priority Review designation from the FDA. Filsuvez Developed by Amryt Pharma,…
My name is Adam Nelson. I am 33 years old and I was born with a rare genetic connective tissue disorder known as epidermolysis bullosa simplex (EBS) of the Weber-Cockayne…
© Copyright 2024 Patient Worthy
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.
