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…
Initially, biopharmaceutical company Anavex Life Sciences ("Anavex") developed Anavex 2-73 (blarcamesine) for patients with Alzheimer's disease. Now, the company has expanded its exploration of the therapy to determine its efficacy…
In some cases, serious complications occur following allogeneic stem cell or bone marrow transplants. For example, in patients with Graft-versus-host disease (GvHD), the donor cells may recognize patient cells as…
Adhik is alive today thanks to the skill and generosity of the Amirita Hospital in India and others who contributed to the cost of using the highly regarded, but…
Aurinia Pharmaceuticals has just announced that Otsuka Pharmaceutical Europe Ltd. (OPEL), their licensing partner, has just filed a Marketing Authorization Application for an investigative therapy for lupus nephritis (LN) called…
I-Mab has just announced China's Center for Drug Evaluation has officially approved the new drug application for felzartamab as therapy for systemic lupus erythematosus (SLE). That means that a Phase…
Alois Alzheimer, a clinical psychiatrist and expert in the study of the nervous system, reported a disease process of the cerebral cortex. The year was 1906. We are approaching July…
BioMan Pharmaceuticals has just announced that vosoritide has just received a positive opinion from the Committee for Medicinal Products in Europe, which means marketing authorization is recommended. Further, it has just…
The FDA grants designations, such as the Rare Pediatric Disease and Orphan Drug designations, to aid in the drug development process. Recently, LEXEO Therapeutics has received these two designations for…
The very first corneal allogenic intrastromal ring segment surgery (CAIRS) for keratoconus has just been completed in Australia. It was completed at Queensland Eye Institute by David Gunn. This procedural…
According to Muscular Dystrophy News, new research suggests that the cholesterol metabolic pathway could offer a potential therapeutic target for patients with Duchenne muscular dystrophy (DMD). Following microRNA analysis, researchers…
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…
When I think of anxiety, I think of the movie, Gremlins (1984). For those of you who have seen this movie, you know that feeding a gremlin after midnight turns…
Patient Worthy was a media partner at this year's NORD Living Rare, Living Stronger Patient and Family Forum. This is an annual event hosted by the National Organization for Rare…
It's summertime, and that means tick season is upon us. We've all had to deal with the annoying insects, whether it's pulling them off our pets, spraying bug spray before…
Within the United States, the FDA developed the Orphan Drug Act to incentivize companies to develop drugs and biologics intended to treat patients with rare conditions. In this case, "rare"…
As reported in Physicians Weekly; a new study wanted to further characterize Enthesitis-related arthritis (ERA), a subtype of juvenile idiopathic arthritis (JIA) that is often is accompanied by anterior uveitis,…
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…
In a story for the Urology Times, a panel of experts on the treatment of non-muscle invasive bladder cancer that is not responsive to Bacillus Calmette-Guérin (BCG) (which is commonly…
We're learning more and more about COVID-19 every day. One thing that we're discovering as the pandemic continues is the complications that may arise after recovery. Chronic fatigue syndrome, brain…
Clinical trial data is extremely helpful in understanding the safety, efficacy, and tolerability of various potential treatments. According to Muscular Dystrophy News, top-line data from the Phase 2b VISION-DMD…
Anybody who knows me knows that Jeopardy! is one of my favorite shows (Rest In Peace, Alex Trebek). Of course, that doesn't mean that the show never messes up or makes…
In a video posted on Healio, Dr. Lorenzo D'Antiga from the Pappa Giovanni XXIII Hospital talked about the findings from a recent clinical study in which the drug odevixibat (also…
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