According to a story from news.cision.com, the US Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for CS1, an investigational drug being developed by the…
After a case of the rare tick-borne illness, Powassan virus, was reported in Providence, Rhode Island in June, Patch released an article detailing the different diseases ticks can carry, how…
According to a press release from Business Wire, the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recently issued a positive opinion in regards to the…
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…
ABC News recently featured an article about Isabella Cristobal and Sergio Soto. They decided to get married shortly after learning that Sergio’s cancer had spread and he had only…
Part II And Finally, Maguire and colleagues also reported on the care of NAS infants and found three areas somewhat similar to the last two reports. The team reported concern…
Part I This is the first of a two-part series on ‘compassion fatigue' and burnout in an area of nursing that, over the years, has seen a significant increase in…
According to a recent article published in The Online Trading Mentor, Inventiva S.A. (Inventiva) has recently opened enrollment for its Phase 3 trial of Lanifibranor, an investigational treatment for nonalcoholic…
My daughter, Carson, was born in June 2020. I received a call 1.5 weeks later about Carson’s abnormal newborn screening. I was told it was most likely an error, but…
There are so many fun activities to do in the great outdoors, especially when the weather is nice. Whether it's going on a scenic hike, having a picnic in the…
According to a story from ANCA Vasculitis News, the biopharmaceutical company ChemoCentryx has recently made an amendment to its approval application for its investigational therapy. This therapy, called avacopan, is…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat patients with rare or life-threatening illnesses, defined as those affecting under 200,000 people or…
Genetic Engineering and Biotechnology News recently published an article describing how engineered cells were implanted into rheumatoid mouse models. The cells used in the study were able to sense…
In a recent press release, the US Food and Drug Administration (FDA) has authorized a third dose of the Moderna or Pfizer COVID-19 vaccine for people living with compromised immune…
Believe it or not, it wasn’t until final exams my freshman year in college that I heard the term “stressed out.” It just wasn’t used that often back then. Nor…
Steven Rodriguez was nineteen years old attending college in California when he began to have severe headaches. A recent article from the UCSF Science Center gives an account of Steven’s…
The outcome of myelodysplastic syndrome (MDS) varies. The disease can lie dormant for decades, or it can become fatal in a matter of months. According to a recent article in…
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…
September is Charcot-Marie-Tooth disease (CMT) Awareness Month! The Charcot-Marie-Tooth Disease Association has put together 5 different ways you can help spread awareness of this rare disease this month. The Association…
Written by Gina Baker This year Global Mitochondrial Disease Awareness week is September 19-25, but my family and I will continue to fight this battle for the rest of…
There are now plans in place to study a gene therapy for GNE myopathy (GNEM), with human dosing expected to begin in 24 months. The plans were developed by the…
According to a recent article at MSN.com, chronic fatigue syndrome is finally receiving the attention it needs after long COVID sufferers experience similar symptoms. Myalgic Encephalomyelitis (ME) Myalgic encephalomyelitis…
In the United States, Fast Track designation is granted to help expedite drug development and review for serious, rare, or life-threatening conditions. To receive Fast Track designation, the treatment must…
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According to a recent article at EverydayHealth.com, wet age-related macular degeneration (wMD) does not have to signify the end of a patient’s career. With developing treatments and tips from specialists,…
Living with an invisible illness can be difficult for a myriad of reasons, one of which is awareness. Those affected by vestibular disorders know this all too well. The word…
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