An article that appeared recently in Psychology Today asks whether dementia is responsible for falls. The answer brings to mind the many medical and environmental factors that affect the elderly…
September is recognized as Charcot-Marie-Tooth disease (CMT) Awareness Month. Although this illness is one of the most widespread inherited genetic disorders, there is still a significant need to spread awareness…
According to an article from ScienceBlog, a twist on CRISPR gene editing has allowed a team of researchers to successfully treat myotonic dystrophy type 1 in mouse models. By modifying…
According to a story from BioSpace, Ziopharma Oncology, Inc. has recently announced that its investigational therapy Ad-RTS-hIL-12 with veledimex (also called Controlled IL-12) has been given Rare Pediatric Disease designation…
Mononine, a treatment for hemophilia B, hit the market nearly three decades ago in 1992. Since then, there have been amazing advancements in the knowledge and treatment for this disease.…
According to a story from BioSpace, the biopharmaceutical company Clene Nanomedicine, Inc., has recently announced that it has completed the process of enrollment patient participants in its upcoming phase 2…
According to a story from BioSpace, the biopharmaceutical company Zogenix, Inc., has recently announced the release of results from a phase 3 clinical trial testing the company's drug fenfluramine (marketed…
As reported in BioSpace; today, Biocryst Pharmaceuticals has been given a boost to further their work to explore the merits of a potential oral treatment for PNH. The U.S. FDA…
According to a story from BioSpace, Takeda Pharmaceutical Company Ltd. recently announced the release of results from a phase 3 clinical trial. This trial was testing the company's drug ixazomib…
This year, the Digital International Liver Congress took place online from August 27-29, thanks to COVID-19. During the conference, biopharmaceutical company Mirum Pharmaceuticals announced data from its Phase 2 INDIGO…
According to a story from Newswise, September is recognized as Pulmonary Fibrosis Awareness Month. This includes idiopathic pulmonary fibrosis (IPF), or disease without an identifiable cause. In the midst of…
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…
According to a story from BioSpace, Genentech (of the Roche Group) has announced its plans to present new data related to its drug satralizumab-mwge (marketed as Enspryng™) at the upcoming…
As reported in Biospace; You probably know that a purple ribbon is used to commemorate cancer, pink for breast cancer, orange for leukemia. What do you know about childhood cancers…
According to Charcot-Marie-Tooth News, patients with Charcot-Marie-Tooth disease type 1A (CMT1A) have an extra PMP22 gene. However, new research shows that this excess gene causes a toxic accumulation of protein within cells.…
Although we are now months into the global pandemic, new information emerges daily. Currently, COVID-19 is responsible for over 29 million cases globally, with 926,000 associated deaths. In the United…
According to a recent article in Genetic Engineering and Biotechnology News, the neural circuit that is responsible for insomnia has been identified by scientists at the Cold Spring Harbor Laboratory…
When I was 12 years old, following my mom’s sudden cardiac arrests, I was diagnosed with a rare, genetic sudden arrhythmia death syndrome called congenital long QT syndrome type 5.…
There is currently an unmet medical need for therapies and research for Parkinson's disease, with the incidence increasing drastically. In fact, the number of cases around the world is expected…
As reported in Science Daily, researchers at the University of Bristol’s MRC Epidemiology Unit working with pharmaceutical companies have developed an approach to reduce the failure rate of developing drugs.…
According to a story from Medscape, thyroid storm is a collection of symptoms that can be described as acute hyperthyroidism (overactivity of the thyroid gland) or thyrotoxicosis, a condition of…
September 15th, 2020 is recognized as World Lymphoma Awareness Day. This day is dedicated to spreading awareness about lymphoma and falls in the context of Blood Cancer Awareness Month, which…
Arguably, our entire year has been dominated by discussions of COVID-19, a novel coronavirus resulting in 28.3 million cases and 911,000 deaths across the globe. In the United States alone,…
Early this week, clinical-stage biopharmaceutical company Enterome announced that the first patient was dosed in a Phase 1/2 clinical trial. The SPENCER trial will evaluate EO2401 as a potential treatment…
The third week of September is recognized annually as Mitochondrial Disease Awareness Week. This global event is held in order to increase awareness of mitochondrial disease in the medical community…
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