According to a recent article, the preliminary results of a study suggest that certain oral and infusion therapies may be beneficial for those suffering from fatigue due to myalgic…
Life is filled with endless ups and downs. The first 5 of my ongoing 20 years living with dystonia were pretty much nothing but down times. I experienced the most horrible pain…
Pursuing an education can be difficult enough without any other extraneous factors. But what if you were also battling frequent infections, gastrointestinal problems, and recurrent hospitalizations? For Evan Brandon,…
X4 Pharmaceuticals Inc. has recently published data from 3 different posters which were presented at the American Society of Hematology (ASH) Annual Meeting in Georgia. You can read the full…
Prometheus Biosciences Inc. has just announced positive results from their Phase 1 investigation for a new treatment for systemic sclerosis-associated interstitial lung disease (SSC-ILD). This trial investigated PRA023, an anti-TL1A…
Back in December, UCB released the results of its phase 3 trial of rozanolixizumab in adult patients with generalized myasthenia gravis (gMG). Fortunately, these data were positive, pointing to this…
Catherine Nettles Cutter, a professor at Penn State University, vividly recalls two years of her life spent trying to recover from a bodysurfing accident that resulted in the mysterious loss…
Over the last 10+ years, there have been extreme advances in the treatment of metastatic melanoma. In particular, researchers have sought to develop therapies which halted cancer progression and reduced…
The Myrovlytis Trust, a UK Charity focused on promoting research and advancing education for rare diseases, and Patient Worthy are now partners! This organization has focused primarily on Birt-Hogg-Dubé syndrome…
According to a recent article, patients with phenylketonuria (PKU) are unable to eat proteins because of their condition. However, a new probiotic might be the solution. Phenylketonuria (PKU) PKU is…
Discerna Pharmaceuticals has just announced the results from a Phase 1 trial investigating their GalXC™ RNAi therapy (Belcesiran) for alpha-1 antitrypsin (AAT) deficiency-associated liver disease (AATLD). The results demonstrated both…
A retrospective medical records study for interstitial lung disease (ILD) has just been published in Arthritis Care & Research. The results found that for women diagnosed with ILD as well as an…
A new study has demonstrated that plasma desmosine could be an acute aortic syndrome biomarker. This also means it could be a potential diagnostic tool. The findings from this study…
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A case study recently reported that an asymptomatic 12-year-old boy was diagnosed with a solitary cutaneous mastocytoma in a rare case. Cutaneous Mastocytosis Cutaneous mastocytosis affects the skin, causing small flat brownish…
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…
2021 was a hectic, record-breaking, and all around stressful year. Now that it's 2022, I think it's time to start the year with a positive story. According to an article…
Just 50 years ago, receiving a diagnosis of severe aplastic anemia could be devastating. While therapeutic options have greatly improved over the past five decades, some doctors have still found…
Fatty Liver Disease This condition is exactly what it sounds like- excess fat accumulates in the liver and causes damage. The condition can have many negative consequences on the body.…
Life with a rare disease can be difficult and scary, especially when you can't be sure what the future holds. Jessica Calkins, a 37-year-old mother from Washington, understands this experience…
One 66-year-old patient who lives in Poughkeepsie, N.Y. received the same diagnosis from three doctors. Each doctor suggested that his symptoms pointed to temporomandibular (TMJ) joint pain. The patient and…
Orphan Drug designation is typically granted to drugs or biologics intended to treat, prevent, or diagnose rare diseases; in the United States, these are conditions affecting under 200,000 Americans. Drug…
After two years of in vitro fertilization treatments, Amber Freed, Age 38, and her husband Mark Freed of Denver, Colorado welcomed their twin babies Riley and Maxwell into the world.…
Equillium Inc. has just announced that their most recent study in lupus nephritis (LN) has been published in the Journal of Clinical Investigation. This study is thanks to collaboration with the Accelerated Medicines…
Due to plastic pollution, microplastics can now be found -- well, nearly everywhere. Microplastics have been found on beaches and in water supplies, in our foods and in the deepest…
Albireo Pharma Inc. has just announced the results from their Phase 1 clinical trial for A3907, an ASBT inhibitor. The study produced positive results, achieving its primary and secondary endpoints. Researchers…
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