In 2020, the U.S. FDA granted Orphan Drug designation to KB407, an investigational gene therapy for patients with cystic fibrosis (CF). More recently, at the start of 2023, the…
Seven years ago, David and Amber Negele married. The pair couldn’t wait to begin their blissful wedded life together. Just a few years after their marriage, David and Amber decided…
From November 10-14, 2022, various stakeholders within the rheumatology sphere connected at the American College of Rheumatology’s (ACR) Convergence 2022. During ACR Convergence, described as the world’s premier rheumatology experience,…
A January 2023 news release from biopharmaceutical company Avidity Biosciences, Inc. ("Avidity") shares that the company's therapy, AOC 1020, earned Fast Track designation from the FDA for the treatment of…
Currently, the standards-of-care for lupus include anti-malarial drugs such as hydroxychloroquine, corticosteroids and immune-suppressive therapies, and biologics such as Benlysta. But new therapies are urgently needed, especially as anywhere between…
Before you read on, make sure to check out Part 1 of Jennifer's story. In Part 1, we discuss what multiple sclerosis is, its symptoms, treatment options, and Jennifer's journey from diagnosis…
The Rogers, Minnesota hockey community is strong and unified; if something happens to someone in their ranks, they step up to assist. But nothing could have prepared them for the…
A few years ago, Dean Mooney began experiencing some troubling health symptoms. His left hand became stiff and sometimes difficult to use. Later, he developed a mild arm tremor. After…
When she was an infant, Phoenix Radziwon was diagnosed with Down syndrome. However, her family has — over more recent years — learned that her Down syndrome diagnosis may go…
Despite medical advances and new therapeutic developments, hepatocellular carcinoma (HCC) remains difficult to treat; in part, this is due to the fact that HCC, like many liver cancers, is often…
According to a story from the Rancho Santa Fe Review, 26-year-old Tyler Schutz began taking the sport of golf by storm last year. The young man made the Double Eagle…
If there is one lesson that Jennifer embodies in full, it’s this: always live life to your fullest. That’s what Jennifer aims to do every day. As an education consultant,…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
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…
James Clarke, now 42 years old, has always loved sports. He watched as Doddie Weir, a Scottish rugby union player, tirelessly worked to raise motor neuron disease (MND) awareness. Doddie…
According to a story from the East Bay Times, it was 2003 when Isabel Bueso and her family traveled to the US from Guatemala. She was just eight years old,…
In the European Union (EU), Orphan designation, or Orphan Medicinal Product designation, is granted to drugs or biologics intending to treat, diagnose, or prevent a rare, chronically debilitating, or…
In March 2022, T. Kavinraj began experiencing a number of worrying health symptoms. He had a splitting headache and could not stop vomiting. In fact, his condition was so bad…
In the United States, the Food and Drug Administration (FDA) grants Orphan Drug designation to drugs or biologics which intend to treat, diagnose, or prevent rare conditions. “Rare” conditions are…
Ten years ago, a biochemist was seeking investors for her new company that was developing mitochondrial-based medicines. The Israeli-born embryologist observed how the blending of mitochondria from one egg…
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…
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 American Society of Hematology (ASH) held its 64th Annual Meeting from December 10-13, 2022. During the meeting, doctors, researchers, and other stakeholders within the hematology sphere came together to…
In multiple disease states, early identification, diagnosis, and treatment can significantly improve overall survival rate and patient outcomes. This is especially true for lip and oral cavity cancer. According to…
Have you ever heard of misophonia? This rare disorder, which literally translates to “hatred of sound,” occurs when certain sounds trigger intense and involuntary emotional and physiological reactions. When people…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
