It’s no secret that receiving a rare disease diagnosis can take excess amounts of time. Various articles share that, on average, it takes anywhere from four to nine years for…
Right now, there are limited pharmacological therapies available for people with nonalcoholic steatohepatitis (NASH). Rather, treatment options focus on healthy diet and exercise, weight loss, alcohol avoidance, lower cholesterol, and…
According to a story from BioPharma Dive, KalVista Pharmaceuticals has halted its clinical trial evaluating an investigational therapy in development for hereditary angioedema. The trial had included a total of…
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,…
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…
The joy of being told you have a normal healthy newborn can at times be overshadowed by concern about whether the baby has a yet-to-be-discovered disease. Medical technology has helped…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is…
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…
Through its recently issued press release, bluebird bio announced the FDA’s accelerated approval of the first therapy to slow the progression of adrenoleukodystrophy (ALD). The disease is the result…
Are you ready for the October 2022 Dysautonomia Challenge? You can make this October stand out on your calendar by making each day meaningful, event ladened, exciting, and dedicated…
On September 28, 2022, the Rare Disease Legislative Advocates (RDLA) hosted their monthly webinar. These webinar help provide updates to the rare disease community on legislation and other policy initiatives…
The young patients began laughing, and some even began to speak and walk. Imagine the excitement among the hospital staff when they realized that a new medication brought about…
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…
This week SciTech Daily published news of the successful eradication of a heretofore incurable form of brain cancer. The research was conducted at Israel’s Tel Aviv University in collaboration with…
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…
According to a story from Undark, Renee Schmidt's first signs of Ehlers-Danlos syndrome became really noticeable when she was a freshman in college. She would experience brief bouts of memory…
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…
Happy Monday! This week, we have stories on a potential treatment approach for an aggressive form of mitochondrial disease, an experimental treatment for osteosarcoma earning Rare Pediatric Disease designation, and…
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