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…
May 7, 2023 will be recognized as Cystinosis Awareness Day. This will be a day to spread awareness about the rare disease cystinosis among the general public and the medical…
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…
Currently, there are no cures for amyotrophic lateral sclerosis (ALS). Symptomatic therapy, alongside disease-modifying treatments like Rilutek, Radicava, and Relyvrio, may be used to manage this condition. However, there…
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…
On April 26th, I had the honor of speaking at the American Brain Foundation’s Commitment to Cures fundraising dinner during the American Academy of Neurology annual convention in Boston. I…
After becoming a de facto football star while playing at LSU, catching 52 passes for a total 629 yards, tight end (TE) Foster Moreau was drafted by the Las…
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…
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…
In July 2022, Patient Worthy reported on data from the Phase 3 REAL 4 clinical trial. During the trial, researchers compared the safety, efficacy, and tolerability of Sogroya (somapacitan-beco)…
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…
As bluebird bio ("bluebird") waited on FDA feedback regarding the manufacturing process for lovotibeglogene autotemcel (lovo-cel) genetherapy, the company disclosed that it would most likely miss its Q1 submission…
Each year, an estimated 20,100 people across America die from non-Hodgkin’s lymphoma (NHL). While treatment is available—namely chemotherapy and immunotherapy—researchers are always exploring new treatment options to help those affected…
A therapy is granted Orphan Drug designation if the FDA believes that this therapy will treat, prevent, or diagnose rare conditions. Rare conditions, in the United States, are those affecting…
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…
The first thing that you should know: osteosarcoma (a type of bone cancer) is treatable. In fact, when you treat osteosarcoma early enough, it is curable; children can live long,…
According to a story from PR Newswire, the biopharmaceutical company Avidity Biosciences, Inc., recently announced that its investigational therapy AOC 1044 has received the US Food and Drug Administration's Fast…
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…
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…
According to a story from Endpoints News, the drug developer Incyte has opted to put an end to its phase III clinical trial evaluating parsaclisib, an orally available PI3K inhibitor,…
Hello, my name is Megan. I have been diagnosed with postural orthostatic tachycardia syndrome (POTS), Ehlers-Danlos syndrome (EDS), mast cell activation syndrome (MCAS), small fiber neuropathy (SFN), eosinophilic esophagitis (EOE),…
Treatment advances are occurring daily in the field of rare disease. As researchers hone their strategies and tools, new technologies are emerging with the potential to significantly impact patients…
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