According to a recent article in Genetic Engineering and Biotechnology News, the blood-brain barrier (BBB) regulates the movement of molecules, cells, and ions between the brain and the blood. Tightly…
As reported in Fierce Pharma; BridgeBio has earned FDA approval for their new targeted cancer treatment, Truseltiq. The approval is the biotech company’s first ever approval for a cancer drug,…
We know that COVID-19 leaves some with long-lasting symptoms. In fact, we are beginning to discover the connection between the respiratory virus and chronic fatigue syndrome (CFS). Now, CFS patients…
Dr. Jerry Vockley, MD, PhD, who currently works as director of the Center for Rare Disease Therapy at UPMC Children's Hospital of Pittsburgh, was confounded. Through his research, Dr. Vockley…
Auto-Brewery Syndrome Auto-brewery syndrome is also known as gut fermentation syndrome. It occurs when fungi or bacteria within the gut produce ethanol by endogenous fermentation. Several different strains may be…
It's no secret that illicit substance use, or substance use disorders, can cause a variety of health effects, including increased risks for certain diseases. As Parkinson's News Today recently reported,…
In patients with rare or underserved conditions, it can sometimes be difficult to receive effective treatment. For example, chemotherapy, the standard-of-care for Langerhans cell histiocytosis (LCH), is only effective in…
Partnerships within the medical community play a huge role in advancing research and developing treatments. According to Charcot-Marie-Tooth News, a new partnership has formed between Dr. Alessandra Bolino, PhD, the…
Newborn screening is important to early diagnosis and intervention. With some conditions, every second counts. Catching a rare disease as soon as a child is born could lead to much…
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"…
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