“Three years ago, my lung function was 11% and I was hours from death, then I received the gift of life – which was Trikafta.” These are the words of…
In December 2022, Liam Hendriks—a pitcher for the Chicago White Sox—was diagnosed with non-Hodgkin’s lymphoma (NHL); he shared this news with the world in January 2023, which Patient Worthy reported…
As reported in Yahoo! Finance, the United States’ Food and Drug Administration (FDA) recently granted Orphan Drug designation to MP1032. This therapy, developed by clinical-stage biotech company MetrioPharm, is being…
In 2014, head coach Jimbo Fisher led the Florida State Seminoles to win the BCS National Championship Game. He’s now head coach of the Texas A&M Aggies, recently deemed by…
Pyxis Oncology is driven to improve the lives of people living with cancer through the development of innovative immunotherapy and antibody drug conjugate (ADJ) therapies. Their treatment PYX-201, a novel…
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 families,…
The Orphan Drug Act of 1983 was designed to facilitate the development of orphan drugs, or drugs intended for rare or life-threatening indications. In the United States, rare conditions are…
At the end of February 2023, workers at the Escanaba Billerud Paper Mill began feeling ill. Their symptoms were reminiscent of atypical pneumonia; some people struggled to breathe. According to…
It can be difficult to spur interest in rare disease drug development. Forty years ago, the Orphan Drug Act passed as a way to incentivize drug development within this space.…
Make sure to read Part 1 of Kandise's story. In Part 1, she discusses some of the symptoms of hereditary multiple exostoses (HME), as well as how she finally reached a diagnosis.…
Ready to read? Before you do, make sure to check out Parts 1 and 2 of Tracy's story. In Part 1, Tracy describes the symptoms that led her to pursue a diagnosis. Part 2…
When Kandise MacLeod was twelve years old, she began noticing various growths and tumors popping up on her bones. These sometimes caused pain or discomfort; in one case, Kandise even…
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…
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…
The American Hematology Society (ASH) held its 64th Annual Meeting in December 2022. During the Meeting, a variety of stakeholders—including physicians, researchers, and industry members—gathered to discuss research, trends, and…
This year, the 13th annual Moebius Syndrome Awareness Day (MSAD) will take place on Tuesday, January 24. Moebius Syndrome Awareness Day, as the name suggests, is an event to celebrate…
“I spent the majority of my life fearing the weight of VHL will eventually crush me, but thanks to this drug trial, not only do the rest of my fellow…
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,…
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…
According to a story from Patient Worthy partner CureDuchenne, the organization has recently launched its CureDuchenne Occupational Therapist Certification Program. This program is intended to give occupational therapists advanced skills…
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…
It’s no secret that being a part of the chronic illness and/or rare disease community can sometimes be lonely and isolating. Many people, while empathetic to the challenges faced within…
According to a news release from biotechnology company Sound Pharmaceuticals, enrollment has begun in the Phase 3 STOPMD-3 clinical trial. So far, the first patient has been enrolled – with…
1On July 27, 2022, global biopharmaceutical company GSK plc shared some exciting news via news release. The company’s therapy Benlysta (belimumab), designed for pediatric patients with lupus nephritis, is now…
The National Organization for Rare Disorders (NORD) recently held its Living Rare, Living Stronger Patient and Family Forum on June 26, 2022. This event took place both virtually and in-person…
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