Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.
According to a late October 2022 news release from gene therapy company AVROBIO, the sixth patient has been dosed in a Phase 1/2 clinical study evaluating AVR-RD-04 for patients…
Before you read on, don't forget to check out Part 1 of our interview with Jennifer Wallace Valdes, PT. In Part 1, Jennifer discusses her background, what Duchenne muscular dystrophy (DMD) is,…
The European Society for Gene and Cell Therapy (ESGCT) recently held its Annual Congress from October 11-14, 2022. During the ESGCT Congress, various stakeholders discussed data, trends, and new research…
The Orphan Drug Act was passed in 1983 to stimulate the development of therapeutics for rare diseases. With this came the Orphan Drug designation. This designation is granted to drugs…
Glioblastoma multiforme (GBM) is notoriously difficult-to-treat. This cancer is both aggressive and invasive, spreading “tentacles” and becoming hard to remove even with surgery. As a result, finding more effective therapeutic…
The FDA created the Rare Pediatric Disease Designation and Priority Review Voucher Program to stimulate the development of therapies intended to treat, diagnose, or prevent rare pediatric diseases. These are…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, prevent, or diagnose a rare disease or condition. Rare conditions are those affecting fewer…
According to ABC News, cases of Vibrio vulnificus (V. vulnificus) infections have risen in Florida following the devastating effects of Hurricane Ian. V. vulnificus infection, which may also be referred to as vibriosis, is…
Occupational therapy for individuals with Duchenne muscular dystrophy (DMD) requires a unique and specialized approach to maximize benefit and reduce harm. It is incredibly important that those performing occupational therapy…
Did you know that November is National Family Caregivers Month? Celebrated annually, this month is designed to recognize the contributions of caregivers and honor their actions. In a recent…
At 29 years old, Morgan Nuchols is living the fullest life that she can. She loves challenging herself by doing puzzles. But her favorite activity? Making birthday and holiday cards…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions. A rare condition is one affecting fewer…
Glioblastoma is notoriously difficult to treat and manage. The cancer is not only aggressive and quickly spreading, but is often treatment-averse. Because of this, glioblastoma comes with a poor prognosis…
For the last few decades, there has been increased interest in understanding the effects of sleep (or lack of!) on our overall health. A recent study sought to understand the…
Before you read on, make sure to check out Part 1 of our interview with Geraldine Bliss, the President and Co-Founder of CureSHANK and mother to Charles, who has Phelan-McDermid syndrome. Today,…
Prior to her daughter Avenly’s diagnosis, Lacy Biram had never before heard of Phelan-McDermid syndrome (PMS). Currently, there are an estimated 2,200-3,000 people worldwide who have this rare condition. For…
On November 8th, 2022, CureSHANK is sponsoring an Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting for Phelan-McDermid Syndrome (PMS). Co-planned by the Phelan-McDermid Syndrome Foundation, this virtual EL-PFDD meeting will: [give]…
Gonzolo and Jennifer Garcia were so excited when they learned that they were pregnant with their son, Asher. But during the 20-week ultrasound, they found out that Asher would be…
Branson Figueroa is four years old – and he’s tough. He has to be; after being born with craniosynostosis, Branson has had to undergo skull reconstruction surgeries twice already. According…
According to the Institute of Cancer Research (ICR), a collaborative pilot study spearheaded by the Institute found that liquid biopsies could aid in the management, care, and treatment of…
According to a news release from the U.S. Food and Drug Administration (FDA) in early November 2022, Dupixent (dupilumab) is now approved for the treatment of adults living with…
Unfortunately, there are no cures - nor effective treatment options - for individuals with P-subtype small cell lung cancer (SCLC). This subtype is notoriously treatment-averse; it is also relatively…
Laura Brown never planned on running more than one marathon. In 2012, she tackled a marathon as a way to cross an item off of her bucket list. But since…
When the COVID-19 pandemic began, many people wondered how it would impact them, especially if living with a rare disease or condition. As the pandemic has progressed, more research has…
Unfortunately, not much is understood still about chronic traumatic encephalopathy (CTE). However, we do know that it has been linked to repeated head trauma or head injury, such as that…
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