In this digital age, rare disease issues have become more prevalent and more visible. According to Eurordis, a rare disease affects less than 1 in 2000 people. There are more…
Many Twitter users were confused and concerned when Julie O'Mahony posted a picture of her mother's hand with two of the fingers white and completely drained of color. However, while…
On April 28th, the virtual World Orphan Drug Congress USA 2021 was held. The program featured a variety of subjects relevant to the development of orphan drugs and the rare…
In the last decade, scientists have made great strides in uncovering details of the underlying causes of neurodegenerative diseases. It is only a matter of time that treatment and prevention…
Ryan Alam was diagnosed with mitochondrial-membrane protein-associated neurodegeneration (MPAN), a rare form of neurodegeneration with brain iron accumulation (NBIA), but he hasn't let these conditions slow him down. He has just…
Gene therapy is a recent development in the industry, and it is one that has brought hope to many people who live with a genetic condition without a viable treatment…
According to a news release from April 2021, a new partnership is working to improve the commercialization of rare disease drugs for patients in China. Together, rare disease therapeutics company…
The Rare Disease Legislative Advocates (RDLA) held its monthly webinar on April 22, 2021. In this informational webinar, the organization featured several speakers who provided updates on some of the…
Peripartum cardiomyopathy is a rare heart ailment, characterized by a weakened heart muscle, that occurs during pregnancy. But why do some pregnant women develop this condition while others do not?…
Happy Friday! Did you know Patient Worthy has a podcast? In the latest episode, we discuss rare disease dermatology with Dr. Steven Chen, MD of Harvard Medical School. Pemphigus and…
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A decade ago, Rob Long's dreams were centered solely on football. He was looking forward to the NFL draft after a successful college career at Syracuse University, but unfortunately, a…
Alarming details of Katy Grainger’s sepsis infection were covered in the April 2021 issue of Women’s Magazine. The interview took place over two years after Katy lost both feet and…
Scholarships are being provided to rare disease patients, thanks to the EveryLife Foundation for Rare Diseases. The scholarships are each worth $5,000 and are intended for those seeking an education…
Prime editing, a new entry in gene-editing technology, is said to be an extension of the “genetic toolbox.” According to a recent article in Science Daily, prime editing designs…
For small patient groups, creating momentum towards rare disease solutions often seems futile. To tackle such expensive and extensive endeavors, rare communities find strength and hope by putting their minds…
C. difficile is a bacterial infection that impacts the gastrointestinal system. It can lead to weight loss, diarrhea, dehydration, and even the loss of blood platelets. Megan Newman has had…
Sawyer Burch is a four-year-old boy from the Nashville, Tennessee area who was born with trifunctional protein deficiency, a rare disorder that takes the body's ability to utilize certain types…
Severe Combined Immunodeficiency Severe combined immunodeficiency (SCID) is a group of diseases caused by mutations that affect how the immune system works to fight infections. These anomalies are usually passed…
With genetic disorders, there is always a big question: do specific gene mutations respond differently to treatment? How can treatment be targeted to best suit the needs of patients? According…
The rare liver disease primary sclerosing cholangitis (PSC) may impact just .01% of people, but the extremely pernicious disorder's attacks on the liver mean bearers of the disorder account for…
Each year, researchers make new strides into genetic research, including the discovery of new genetic disorders. According to Medical XPress, researchers from the University of Portsmouth and the University of…
Rare disease patients throughout India have expressed their disappointment and anger towards the country's new policy centered on rare conditions. One of their main concerns is that the policy changes…
On April 2nd, NFL scouts made their way to Eugene, Oregon to check out some of college football's most talented players. They watched as athletes completed the vertical jump, 40-yard…
Cancer Network recently reported that Jazz Pharmaceuticals’ Vyxeos was approved by the FDA to treat therapy-related pediatric patients one year or older who were newly diagnosed with acute myeloid…
Financial Burdens in Rare Disease In 2019, 20% of all adults in the United States have reported that they had large medical bills that were unexpected. 18% currently had medical…
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