On July 28, 2022, the Rare Disease Legislative Advocates (RDLA) hosted their monthly webinar for July. As always, these recurring meetings help provide critical updates to the rare disease community…
When Ryker Colón was first born, his parents Eddie and Brandi noticed something potentially concerning. Their young son had a chronic cough - and nobody could seem to figure out…
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…
Written By Ashely Winslow, PhD, President & Chief Scientific Officer of Odylia Therapeutics One of the biggest issues impacting many rare disease communities is the availability of approved treatments. While…
Although many people in the United States face challenges when seeking healthcare services, new research has found that when seeking a diagnosis for a rare disease, the situation is…
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…
According to a recent article, a man who developed terrifying symptoms following a stroke was found to have statin-associated immune-mediated necrotizing myopathy. Unexpected Symptoms After Stroke When her…
The novel program called BeginNGS™ was described in a recent article which has a goal of testing for five hundred disorders and several thousand infants. Rady Children's Institute for…
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…
Getting a rare disease diagnosis can be difficult and take a long time. This can be a problem, as time is something that those with progressive illnesses don't have much…
According to a recent article, a new study where SIOD patients had a dual immune/solid organ transplant yielded positive results with patients accepting their new kidneys without rejection. Schimke Immune-Osseous…
According to a recent press release from Biospace, the first cohort of the ReSPECT-LM trial has officially completed enrollment. This trial focuses on Rhenium-186 NanoLiposome (186RNL) as a treatment for…
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science…
Meghan Rourke saw the need for more open conversations about chronic illnesses, sparking her to write The Invisible Kingdom: Reimaging Chronic Illness. In an excerpt from her novel, she shares…
Jonathan Rayner works at the University of South Alabama as the Director of the Laboratory of Infectious Diseases. Jonathan has studied ticks since back in 2018. He and his colleagues…
According to a recent article, a 6(b) study of pharmacy benefit managers and anticompetitive business practices was approved by the Federal Trade Commission following pressure from the NCPA. The Study…
I've known some wonderful fathers throughout my life. My own father was a gem. My siblings agree. However, it was not until I became immersed in the rare disease world…
At the end of May, 2022, the Governor of Connecticut, Ned Lamont, established a permanent Rare Disease Advisory Council (RDAC) for the state by signing House Bill 5500 (HB 5500).…
In the United States, a rare disease or condition is one which affects fewer than 200,000 individuals. Alternately, a rare condition in the European Union (EU) is considered one which…
Personable. Loving. Impactful. Those are all words that Angie Kane would use to describe her son Colton, who passed away at 21 years old in October 2021. When Angie talks…
We have been told occasionally that certain phrases we use when speaking with a terminally ill patient may sound insensitive. We have also been told what we should not say…
Advocacy is an extremely important part of supporting and helping the rare disease community. A big part of this is rare disease advisory councils (RDACs), which are governmental groups comprised…
As we know, many treatments for rare diseases are quite expensive - and this price doesn't even factor in travel, lodging, appointments, and more. To help with this, the government…
Sydney Miller, born in the UK in March 2020, has an extremely rare disease. Newsweek recently carried a story about Sydney and her illness. This little girl captured the hearts…
It is very rare that one might say that a car accident saved their life – but Yolanda Carusciello, who lives in Brooklyn, can say just that. Of course, when…
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