When news of a bacterial meningitis outbreak spreads, you may hear the phrase "cluster." When referencing a small cluster, it refers to a smaller group of people, often in a…
According to a story from Rolling Stone, iconic comedian Gilbert Gottfried has passed away from complications related to myotonic dystrophy type II, a rare disease. His publicist disclosed his cause…
At the end of March 2022, ALS News Today announced that IFB-088, an experimental treatment for amyotrophic lateral sclerosis (ALS), earned Orphan Drug designation from the FDA. The treatment was…
Make sure to check out Part 1 of our interview with Dan Pezzetta, an advocate for the rare disease and chronic illness communities. In Part 1, we dug into Dan's…
The rarity of certain diseases often stops them from having numerous treatment options, and the variability of patient experiences makes it so that each option does not necessarily work for…
According to a recent article, the treatment for macular edema associated with uveitis, Xipere, is now available in the United States. Uveitis Uveitis is a term that refers to inflammation…
A recent study has evaluated the incidence of thromboembolic events (TEE) in patients diagnosed with advanced HR-positive, HER2-negative breast cancer. Specifically, researchers were concerned that treatment with cyclin dependent kinase…
In a late March 2022 news release from biopharmaceutical company CANbridge Pharmaceuticals, Inc. ("CANbridge"), the company noted that the first patient was dosed in a Phase 1b/2 clinical trial evaluating…
In 2020, the CDC estimates that there were 241 million cases of malaria globally, of which 627,000 people died. Unfortunately, many of the mortalities were associated with children in sub-Saharan…
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…
Professors Susan Treves and Francesco Zorzato, Basel University researchers, have recently presented the first potential approach to congenital muscle diseases. Their report was published in a recent article by Science…
A recent study has examined the characteristics and the clinical outcomes for individuals with severe anti-neutrophil cytoplasmic antibody-associated vasculitides (AAVs). AAVs are a very rare group of systemic autoimmune conditions.…
Currently, no approved therapies exist for ADNP syndrome, a rare genetic neurodevelopmental disorder. But a group of researchers from the UC Davis MIND Institute are working to change that. According…
At 22 years old, Dan Pezzetta is carving out a name for himself in the chronic illness sphere. When he was born, Dan was diagnosed with aortic stenosis. Although intervention…
A clinical trial recently conducted at the Pediatric Imam Hossein Hospital demonstrated that synbiotics (a mixture of prebiotics and probiotics) can significantly improve symptoms for pediatric eosinophilic esophagitis (EoE) patients.…
A retrospective study recently published in the journal Pediatric Rheumatology has detailed the different antiphospholipid syndrome (APS) phenotypes in pediatric patients in effort to develop care approaches which are more personalized. APS APS is…
Celia Grace, a metachromatic leukodystrophy (MLD) patient from Alabama, has recently made history in Minnesota - and she's just five years old! Celia recently underwent an investigational gene therapy to…
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…
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