Community support can be extremely uplifting and important, especially when one is impacted by a rare disease. This is the case for three-year-old Charlie Fry, who was born with SLC6a1.…
A study conducted by researchers at Nagoya University in Japan and researchers from Yale University in the United States was recently published in the European Respiratory Journal. This study utilized single-cell…
Clinical trials can be crucial in determining potential treatment options for a variety of conditions. According to Pharma Biz, biopharmaceutical company Nordic Nanovector ("Nordic") recently shared promising data from the…
It's the season of sun and summer fun, which leads many to enjoy the great outdoors and warm weather. While this is all in good fun, it's important to remember…
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 patient sitting in front of this physician was later diagnosed with severe chronic fatigue syndrome (myalgic encephalomyelitis or ME/CFS). But for now, the patient had to endure humiliation…
Amyloidosis is a rare disease which can cause organ failure. It is the result of an abnormal buildup of amyloid proteins in the blood. These proteins are then deposited within…
In a study published in the research journal Scientific Reports, CD71+ erythroid cells (CECs) have an immunoregulatory function. They often appear in people with cardiopulmonary or hemolytic disorders, as well as in…
Dr. Jerry Vockley from the UPMC Children's Hospital of Pittsburgh has been studying the rare disease VLCAD for years. His work has led to the creation of two therapies which…
Salarius Pharmaceuticals has just announced the addition of a new clinical trial site for their study investigating seclidemstat as a treatment for Ewing sarcoma (relapsed or refractory) as well as…
Even though some treatments are beneficial for patients, these same treatments can also cause adverse reactions. This seems to be the case for Isturisa (osilodrostat), according to Cushing's Disease News.…
33 year old Emily Helm from Brandon, Mississippi, had always learned that many of her family members on her father's dealt with bone weakness, joint problems, and pain. It was…
Acknowledgment: This story is sponsored by Takeda and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing relevant, vetted and valuable information to the…
As we've learned more about individuals facing long-term symptoms from COVID-19, we have heard more and more about how it mimics other chronic conditions. For instance, those suffering from chronic…
Many individuals in our day and age find it difficult to sleep. Although the first humans awoke with the sun and slept with the moon, we don't operate that way…
From July 2-3 and 8-10, 2021, the European Crohn's and Colitis Organisation (ECCO) held its 2021 virtual Annual Congress (ECCO'21). During the Congress, many discussed new research and trends…
Chronic Fatigue Syndrome Chronic fatigue syndrome (CFS), otherwise known as myalgic encephalomyelitis (ME), is a rare disease primarily characterized by unexplainable and intense fatigue that is not relieved with rest.…
The healthcare system is designed to provide safe, effective, and beneficial care for all. Unfortunately, the current system represents a wealth of socioeconomic and racial disparities. Past studies have shown…
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…
Seven years ago, when Hunter Parrick was 10 years old, he went for what could've been a routine ear surgery. However, during the surgery, doctors found out that Hunter was…
A new clinical trial is starting for Friedreich's ataxia (FA). This trial will investigate if supplementation of vitamin D in its active form, also known as calcitriol, will aid in…
For patients with Cushing syndrome, transsphenoidal surgery can be a beneficial option for addressing pituitary growths or tumors. But according to Cushing's Disease News, this same surgery also increases the…
In the United Kingdom, the Medicines and Healthcare products Regulatory Agency (MHRA) regulates medication, transfusion components, and medical devices, making sure they meet safety and efficacy standards. According to Pharma…
Clinical trials are important research tools to help understand the safety, efficacy, and tolerability of potential treatments. According to a news release from July 19, 2021, biotechnology company Prometheus Biosciences,…
Access to treatment is an issue for patients across the world, especially for those with rare diseases. Laurie Young, who was diagnosed with type 2 spinal muscular atrophy (SMA) at…
© Copyright 2024 Patient Worthy
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.
