Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
For the first two months of her pregnancy, Kristine Koser felt pretty good. Koser and her husband, Andrew, couldn't wait to welcome their daughter Aubrey into the world. Everything seemed…
When Tom and Tammy Parteleno first learned that their son Michael had Batten disease, it felt overwhelming. Scary, even. What would the future bring for their son? The Parteleno family…
A few words of comfort from NINDS, the National Institute of Neurological Disorders and Stroke as featured in MSN. This article is designed to help you cope with the pain,…
Lynch syndrome is a condition that increases the predisposition for certain cancers. People with Lynch syndrome should undergo frequent cancer screening for early diagnosis and treatment. But if Lynch syndrome…
It can be frightening and overwhelming to receive a rare disease diagnosis—and especially so if that rare disease is still poorly understood. When the Borofka family learned that their son…
Throughout her seventeen years alive, Daisha Felps has learned to face obstacles with resilience and strength. And obstacles come around more than she would like. You see, Felps has sickle…
Oakland A's Game Day Uplifting Experience September 15, 2023 Alameda County Coliseum, Oakland, CA Uplifting Athletes is excited to invite rare individuals and families to join us for a memorable…
617 words (source - 3% match) vs. 452 words (mine - 4% match) As our healthcare field continues to innovate and grow, we have seen more conversations regarding gene therapy…
As many people in the rare disease community know, it can be difficult at times to galvanize support for improved drug development. Many larger pharmaceutical companies may be hesitant to…
The ocean is my favorite place in the entire world. When I see it—its immensity, its beauty—I feel immediately at home in the world. I believe that everyone should have…
According to a story from The People's Pharmacy, a Senate report from 2018 highlights how opioid manufacturers regularly donate large sums of money to professional organizations and patient advocacy groups.…
Previously approved for the treatment of narcolepsy, pitolisant (marketed under the brand name Wakix) may soon be leveraged for another rare condition: idiopathic hypersomnia (IH). Pitolisant is a histamine 3…
Also known as Richter’s Transformation, Richter syndrome is a rare and aggressive complication of chronic lymphocytic leukemia (CLL). When someone develops Richter syndrome, their CLL can transform into another rare…
A 15-year search for a diagnosis by Lilly Grossman and her parents ended in 2013. Lilly became the first person in the world to be diagnosed with ADCY5-related dyskinesia.…
An Intern’s Journey When my summer internship with Red Nucleus began, I had little familiarity with rare diseases and the life science industry. A distant relative of mine struggled with…
Unfortunately, gaining access to care within the rare disease space can be difficult. There is often lesser education and awareness about rare conditions, less research performed, and poorer access to…
21-year-old Chloe Feighery has a deep love and appreciation for her younger sister Heidi, who is just one and a half years old. When she learned that she was becoming…
Soon after Emma Young was born, she was diagnosed with a rare genetic disorder called Bannayan-Riley-Ruvalcaba syndrome (BRRS). At two months old, she battled COVID-19. Then she was diagnosed with…
For the first year of his life, Warner Kays looked like an average, healthy, happy baby. He crawled around, took joy in family time with his older sister Presley, and…
For many people, a broken bone would severely interrupt their life. But six-year-old Avery Balcazar has learned to live with it—even when it’s tough. As reported by CBC News, Balcazar…
Sounds like ‘food for thought’. The term ‘sandwich generation’ describes family members who are usually between the ages of 45 and 55 and are responsible for raising their children…
Authored by Dr. Harsha Rajasimha, Founder and Executive Chairman, IndoUSrare. Co-Authored by Dr. Padma Rammoorthy, Medical Consultant, IndoUSrare. The diagnostic journey for patients with rare diseases often becomes a prolonged odyssey,…
The number of novel drugs approved by the FDA has doubled in the ten-year period from 2012 through 2020. Investment in investigational drugs for rare diseases appears to be continuing…
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…
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