November 1st marks Lennox-Gastaut syndrome (LGS) Awareness Day, which is dedicated to patients living with this severe epilepsy syndrome. The LGS Foundation has organized this day to celebrate the resiliency…
Health officials in Long Island, New York are investigating ten reported cases of Legionnaires' disease, which is a severe form of pneumonia. While medical teams are searching the area for…
We know to be wary of ticks during the summer; it's when we spend the most time outdoors and the weather is at its best. But did you know that…
According to an article recently published in WhoTrades, Dupixent has recently been assessed in a clinical trial for the treatment of uncontrolled prurigo nodularis in adult patients. The data were…
Climate change is an issue that has seen a lot of activism from the younger generations, as they will have to grow up and handle more of the burdens it…
Collaboration can be extremely important when it comes to developing treatments, especially for rare disorders. Sharing information, technology, and expertise can lead to therapies that otherwise might not have seen…
Currently, there are no FDA-approved treatments for nonalcoholic fatty liver disease (NAFLD) or its more advanced version: nonalcoholic steatohepatitis (NASH). This is partly due to the lack of understanding surrounding…
Currently, about 90% of cystic fibrosis (CF) patients have a treatment option. This percentage jumped from 50% to 90% upon the approval of Trikafta. However, the remaining 10% of CF…
A new ale is hitting Doubleday’s and The Bit in Dobbs Ferry with a very special purpose. Named "Jacob the Warrior" IPA, this beer was created to raise awareness for…
Rats are a part of New York City - if you've ever been, you've definitely seen them. Most of the time, they're just pests on the streets or subway that…
A new partnership has been formed between Global Genes and the Rare Disease Diversity Coalition (RDDC) - created by the Black Women's Health Imperative - in an effort to increase…
A lack of awareness is a problem that many rare diseases face. Small patient populations, limited research, and other factors all play a role in this issue. Luckily, there are…
Atara Therapeutics has released data from its Phase 1 open-label extension (OLE) trial of ATA188, a treatment for progressive multiple sclerosis (MS). This allogeneic T-cell immunotherapy targets B cells and…
The 37th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) was held last week from the 13th to the 15th. This event is the largest…
Now that it's officially October, it's time to celebrate my personal favorite holiday - Halloween. That means haunted houses, scary movies, creative costumes, and spooky decorations. One Transcona family is…
Two and a half years after announcing its launch, Dyne Therapeutics held its very first Research & Development Day. Throughout the day, there were presentations focusing on the development of…
Rare disease therapies are often difficult to access, whether that is due to price, a patient's location, or another factor. Because of these obstacles, many rare disease patients find themselves…
Last week marked National Trigeminal Neuralgia Awareness Day, which was celebrated on October 7th. It's meant to raise awareness of this rare disease while providing support to those affected. You…
Fabry disease patients may have another treatment option soon, according to a press release published in BioSpace. Protalix Biotherapeutics and Chiesi Global Rare Diseases have provided an update on the…
The first patient in a Phase 2a trial of BOS-580 as a treatment for nonalcoholic steatohepatitis (NASH) was recently dosed. Boston Pharmaceuticals, the developer of the drug, aims to address…
Explaining something as complicated as a rare disease to a child can be difficult. That's why Alexion Pharmaceuticals created a books series titled "Inspired By." The series is a way…
A rare disease diagnosis can change your life; Mari Jackson knows this firsthand. She received a surprise diagnosis of pulmonary arterial hypertension (PAH) at age 48. Her symptoms began suddenly,…
According to a recent press release from Horizon Therapeutics, UPLINZA, the only FDA-approved treatment for AQP4 antibody-positive neuromyelitis optica spectrum disorder (NMOSD), may be a viable treatment option for African…
Clinical trials face many obstacles regardless of which disease they aim to investigate. However, rare disease trials face additional problems that don't typically impact studies for more common conditions. Small…
"Rare" is a documentary that gives its viewers a glimpse into the world of rare diseases, something that many people know little about. Until now, the project has been funded…
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