For many rare diseases, gene therapy, not traditional therapeutics, is perhaps the only means to significant relief and cure. However, the path to gene therapy has been strewn with obstacles…
Canada’s unemployment rate is at a record low. Industries have recovered 126% of the jobs lost by the pandemic. On the health front, scientific knowledge and the ability to…
Clinical trials are often mentioned as an afterthought when discussing the length of time it takes for drugs to be approved by the FDA. On the contrary, clinical trials are…
Prasanna Shirol, Executive Director of the Organization for Rare Diseases India (ORDI), expresses his opinion to the Times of India about the urgency to address the issue of rare…
According to an article published in Cold Agglutinin News, Dianthus Therapeutics (“Dianthus”) is planning on holding a proof-of-concept study in 2024. Within the study, the company would evaluate the safety…
People with severe cases of Eastern equine encephalitis (EEE) or Venezuelan equine encephalitis (VEE) can develop significant brain inflammation, causing damage. An estimated 33% of people with severe EEE infection,…
Have you been struggling with gastrointestinal (GI) issues? Does your stomach ache? Have you been throwing up or experiencing diarrhea—but you’re not quite sure why? Well, you might have alpha-gal…
In 2021, Josie Gosney entered the world and her parents couldn’t be more thrilled. When she was first born, Josie showed signs that something might be right. But after a…
Usher syndrome is a rare genetic disorder that is characterized by deafness and often paired with retinitis pigmentosa (RP), an inherited disease which causes retinal degeneration and vision loss. There…
NORD's Living Rare, Living Stronger Patient & Family Forum May 6, 2023 featuring the Rare Impact Awards (May 4) Hosted at the Renaissance Downtown Hotel, we will bring the rare disease…
The London Marathon took place on April 23, 2023—and one mom was running for an especially important cause: to raise Angelman syndrome awareness. According to an article from the BBC,…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…
Collaboration has always made the world go round - and it's no different in rare disease research. Sometimes, the community affected by a certain rare disease is small, which…
When it comes to staying active, 8-year-old R.J. Walters has it covered. His favorite sport right now is basketball. But that hasn’t stopped R.J. from getting his yellow belt in…
Dear fellow rare community, It is with great joy that I share my journey of self-discovery and self-love with you. It took me a while to fully embrace my uniqueness…
Altogether, there are an estimated 300,000 people in Ireland who are living with a rare disease. However, given the increasing amount of genetically-oriented rare diseases, it's possible that even…
On April 20, 2023, the Rare Disease Legislative Advocates (RDLA) hosted its monthly webinar. These webinars help provide updates to the rare disease community on legislation and other policy initiatives…
In a news release on the company’s website, biopharmaceutical company Applied Therapeutics, Inc. shared that positive data was available from the Phase 3 ACTION-Galactosemia Kids trial. Within the trial, researchers…
Rylee Riekena feels most at home when she’s near water. There’s something about the ocean that just makes her feel safe and at peace. So when she took up paddleboarding,…
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…
Each year, students at Carteret Community College in Morehead City, North Carolina participate in the “Great Strides” Walk for Cystic Fibrosis. “Great Strides” is the Cystic Fibrosis Foundation’s largest fundraising…
Over the years, there has been minimal improvement in the life expectancy differences between white and black people living in the United States. Yet, according to an article featured…
According to a recent study from McGill University in Canada, the new method for classification of brain illnesses has been identified. This study was led by Yashar Zeighami and compared…
On February 1, 2023, FDA issued a Draft Guidance Document, called “Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products” (Docket ID: FDA-2022-D-2983). The Draft Guidance considers…
On April 12, 2023, Research!America, a Patient Worthy partner organization, hosted a webinar program titled Better Data for More Equitable Public Health Research. This talk featured Dr. Mary Pittman, President…
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