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.
Did you know that October is Spina Bifida Awareness Month? This month is designed to celebrate those living with spina bifida, spread awareness and education among the general public,…
According to a September 2022 article from OncLive, the European Medicines Agency (EMA) granted Orphan Drug designation to CAN-2409. This therapy is being developed as a potential therapeutic option for…
For as long as he could remember, Georgie Davey had one word that he thought described himself: “clumsy.” When he was a child, Georgie would frequently trip and fall. He…
Sometimes, it can be difficult to incentivize researchers and drug developers to focus on solutions for those with rare conditions. The FDA created the Orphan Drug Act to overcome…
Don't forget to read Part 1, where we discuss Dr. Maleddu's background, why she chose to specialize in medical oncology, what a desmoid tumor is, and its symptoms, causes, and treatments.…
In June 2022, 16-year-old Patrick McLaughlin was officially diagnosed with aplastic anemia, a rare form of bone marrow failure. Inspired by Patrick's journey and his resilience throughout this process, the…
Don't forget to read Part 1 of our interview. In Part 1, we discussed what chronic granulomatous disease (CGD) is, how it is treated, and how parents and physicians can identify an…
Don't forget to read Part 1 of our interview, where Todd discusses what acute lymphoblastic leukemia (ALL) is and why it is so important to raise awareness for ALL and other cancers. Today,…
September was Desmoid Tumor Awareness Month, a month designed to raise awareness and spread education about desmoid tumors. Additionally, during this month, patients, family members, doctors, and other organizations helped…
In April 2022, the United States approved Ultomiris for the treatment of patients with generalized myasthenia gravis (gMG). This approval was followed, just four months later, by an approval…
For over three decades, Dr. Ben Katz and his research team have been treating patients with chronic granulomatous disease (CGD) and working to build a better understanding of the disease…
Did you know that September is Childhood Cancer Awareness Month? Although September has just passed, it is still important to raise awareness of the many different forms of childhood cancer…
Over the years, there have been growing concerns about medical research failing to cover or explore diverse groups of people. But in a world where healthcare is not just one-size-fits-all, how…
Growing up, Will Kleemeier learned all about polycystic kidney disease (PKD). He had several family members with PKD, so he knew that there was a chance that he too…
It’s October, so you know what that means – it’s Homocystinuria (HCU) Awareness Month! The goal of HCU Awareness Month is to raise awareness of homocystinuria throughout the medical…
Before you continue, make sure you've read Part 1 of our interview, where we discussed the Lord family's story and experience with rare disease, and the development of MobiMedQR. This tool, which…
If you haven't already, don't forget to go and read Part 1 of our interview. In Part 1, David Ogman and I discuss his son Jordan's TECPR2 diagnosis, the diagnostic journey,…
When asked his most important piece of advice for families within the rare disease community, Jeffrey Lord said: Do what you can control and move on from there. Every day,…
In his early stages of life, Austin (A.J.) Rose was diagnosed with an ultra-rare disease called pontocerebellar hypoplasia type 6 (PCH6). Now, at three years old, A.J. is a…
There seems to be no limits when it comes to what a parent will do for their child. And when it came down to it, David and Stacey Ogman knew…
In late September 2022, biotech company Oncoheroes Biosciences, Inc. ("Oncoheroes") shared via news release that its product dovitinib, which the company has an exclusive pediatric licensing agreement for from…
Over the last few years, there have been slight increases in the number of acute flaccid myelitis (AFM) cases throughout the United States. Specifically, 2014, 2016, and 2018 all saw…
In a late September 2022 news release from AVROBIO, Inc., the company’s gene therapy candidate AVR-RD-04 recently earned Rare Pediatric Disease designation. This investigational therapy is being developed as a…
According to an article in Batten Disease News, enrollment is complete for a Phase 1/2 clinical trial. Within the trial, researchers will be evaluating Batten-1 for adolescents and adults with…
Osteosarcoma is genomically complex, which means that – sometimes – it can also be difficult to treat. Treatment and management, however, are crucial to ensuring positive patient outcomes. Doctors and…
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