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.
In the past, it was difficult to incentivize the development of treatments for rare diseases, given issues with both profitability and small patient populations. However, all people are deserving…
Over 30 million people in the European Union (EU) are living with a rare disease. Yet rare disease drug development is still somewhat stifled. Orphan designation helps to incentivize drug…
The most dramatic. Season. EVER!!! If you've ever watched "The Bachelor" or "The Bachelorette," you've probably heard this refrain from the show's host, talking up the ridiculous antics that keep our eyes…
In the past, there have been limited therapeutic options for pediatric patients living with hereditary angioedema (HAE). Children aged 2 to <6 years old had no approved prophylaxis therapy. For…
The 2023 Special Olympics BC Winter Games took place from February 2-4, 2023. Sporting events at Winter Games include 5-pin bowling, curling, figure skating, alpine or cross-country skiing, and more!…
Known as “Slydah,” Chicago White Sox relief pitcher (RP) Liam Hendriks has done amazing things on the field. In September/October 2021, Hendriks was named the American League Reliever of…
For many people living with mantle cell lymphoma (MCL), the standards-of-care include Bruton's tyrosine kinase (BTK) inhibitors. But there is a subset of patients who do not respond well,…
When Arica Svoboda thinks of her two children, Hayes and Hendrix, she can’t help but fill up with love. She tells Patient Worthy: “My boys are special. Their smiles and…
Have you ever heard of Marfan syndrome? This rare inherited disorder affects connective tissue (fibers that support and anchor organs and other bodily structures) throughout the body. Marfan syndrome most…
For many people, a rare disease diagnosis can feel like life is over. For Angel, growing up with homozygous familial hypercholesterolemia (HoFH), a rare genetic condition characterized by extremely high…
Friends and family refer to 41-year-old Teddy Higgins as kind, selfless, and loving. Given all of his support for others, his friends and family are now working to support Teddy…
In a recent news release from biotechnology company Cartesian Therapeutics, the company shared that the first patient was dosed in a Phase 2b study evaluating Descartes-08 for generalized myasthenia…
Treatment for Alagille syndrome, a rare genetic disorder that affects multiple organ systems, are typically symptomatic. Many children born with this disorder eventually require a liver transplant; without this, Alagille…
Neuromyelitis optica spectrum disorder (NMOSD) can have long-term impacts for those affected. Current research suggests that up to 69% of patients may have severe unilateral or bilateral vision loss…
Enzyme replacement therapy (ERT) has been explored as a potential therapeutic option for mucopolysaccharidosis type III A (MPS III-A or Sanfilippo syndrome type A). However, these solutions have not…
What would you do if you were told that your child had a rare disease? How about if your child had an ultra-rare disease, which affects fewer than 20 in…
Over the past few decades, research has shown a potential connection between metabolic disorders and multiple sclerosis (MS). While this connection has been made, anti-hyperglycemic medications (often used to…
When Leonidas Boyne was born, he seemed—for all intents and purposes—to be a healthy, happy child. At five months old, he is still a bubbly, happy baby with a smile…
Before you read on, make sure to check out Part 1 of Emily's story. In Part 1, Emily discusses what Stickler syndrome is and her diagnostic journey. Today, we'll discuss coming to…
There are limited therapeutic options for people living with autosomal dominant polycystic kidney disease (ADPKD); currently, the only real standards-of-care include dialysis and kidney transplantation. When discussing the therapeutic landscape,…
If you’ve ever wanted to run around Philadelphia in your underwear for a good cause, I have an event to tell you about! As first reported by the Philly…
The American Society of Hematology (ASH) held its 64th Annual Meeting in December 2022, during which time a variety of medical stakeholders discussed issues, trends, and research in the…
In the reality television world, Megan Marx is best known for her time on Bachelor Australia, Bachelor in Paradise, and The Challenge Australia. But Marx is about to be…
Living with a rare genetic disease like Stickler syndrome (Or “Sticklers”) isn’t a death sentence. If Emily Katharine wants people to know one thing, that’s it: that they are more…
In a late December 2022 news release from biopharmaceutical company Altimmune, Inc., the company shared that positive topline data was available from a 24-week study—including a 12-week extension—which evaluated…
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