Recently, PFIC Voices released a guide for progressive familial intrahepatic cholestasis (PFIC) patients and families that will help them understand the condition and how they can get support. Progressive Familial…
Elizabeth Dravis is a PhD student studying public health, a medical professional, a runner, and a mother. She also is a minimal change disease (MCD) patient. This is her diagnosis…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Earlier this year, 4D Pharma Plc. ("4D Pharma") experienced a "win" when the FDA approved the company's plans to initiate a Phase 1 clinical trial evaluating MRx0005 and MRx0029 for Parkinson's…
According to a recent article, the results of a new study shows that patients that are diagnosed with Marfan syndrome and an aortic aneurysm or dissection are at a…
25-year-old Grace Strobel is a boundary-breaker. A few years ago, she and her mother began sharing her story at schools or through speeches. You see, Grace has Down syndrome, meaning…
When two previously independent genes join together, and begin transcribing and translating as if they were one, this is known as a fusion gene. Fusion genes have previously been…
Over recent weeks, stories have emerged regarding the frightening situation in Ukraine. People have lost their lives, their homes, and their sources of income. So what happens to those like…
Cushing's disease, like all other rare diseases, benefits greatly from increased awareness and advocacy. Because there are fewer patients and affected individuals, their voices need to be louder in order…
Recent research has given us a better understanding of acromegaly, a rare hormonal disorder. Conducted by Maria Fleseriu, MD, FACE, and colleagues at the Pituitary Center at Oregon Health &…
Did you know that there are six muscles attached to each eye which influence the way the eye moves? When these muscles are properly working, the eyes move together and…
If your life goes awry, do you trust your community to stand by you? Luckily, for 6th grader Ethan Chandler, his Carterville, Illinois community jumped in to help when he…
Since 2015, April 6 has been recognized as Bohring-Opitz Syndrome Awareness Day, a time to spread awareness about this rare disease among the medical field and the general public. The…
Have you ever heard of PD-L1? The National Cancer Institute explains that PD-L1 is a protein which acts as somewhat of a “brake” for immune response. Sometimes, cancer cells will…
CANbridge Pharmaceuticals recently announced via press release that the Taiwan Food and Drug Administration (TFDA) has accepted their New Drug Application (NDA)/Orphan Drug Registration (ODR) for CAN108. Marketed as LIVMARLI,…
It's safe to say that Gabe Barajas has never been afraid of a challenge. In fact, he embraces challenges - and the excitement and adventure they bring to his life.…
Definitive Healthcare launched a survey in November 2021 to help physicians, researchers, and the rare community understand what the biggest challenges are for providing a rare disease diagnosis and treating…
Marine Corps values include abiding by a code of integrity, respecting others, and respecting human dignity. For veteran John Rubino, this code of conduct helps shape the way he lives…
On March 29, 2022, globally diversified chemical company LG Chem announced via press release that its Investigational New Drug (IND) application was approved by the U.S. Food and Drug Administration…
Myrtelle Inc. has just announced that they have been granted three new designations from the FDA for their investigative gene therapy called rAAV-Olig001-ASPA. This therapy is being investigated as a…
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…
Juvena Therapeutics has just announced that they've received a new grant which will fund their research of myotonic dystrophy type 1 (DM1). This was a very competitive grant which totals…
Awareness and advocacy are extremely important to every rare disease, and porphyria is no different. That's why we celebrate Porphyria Awareness Week (PAW) every year from April 2nd until the 9th.…
Currently, there are no cures for idiopathic pulmonary fibrosis (IPF), and the two available treatments aim to reduce lung scarring and symptom progression. However, it is clear that new therapeutic…
According to a recent article, Canadian actor Cameron Mathison opened up about dealing with Legg-Calve-Perthes disease throughout his childhood. Perthes Disease Perthes disease is a condition that affects the hips…
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