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…
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…
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 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.…
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…
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…
According to PharmaField, the Scottish Medicines Consortium (SMC) recently approved and accepted 14 new medicines to be used by NHSScotland. Many of these medications, such as Xospata, are used to…
This year, September 13-19 is being recognized as Balance Awareness Week. Organized by the Vestibular Disorders Association, the goal of this campaign is to help spread awareness among both 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…
The SLEEP 2020 Annual Meeting will be held virtually due to the COVID-19 pandemic, but that is not stopping Axsome Therapeutics from presenting the results of their Phase 2 CONCERT…
The Virtual Vestibular Conference September 14-18, 2020 In conjunction with Balance Awareness Week, the Vestibular Disorders Association will be hosting its first annual Virtual Vestibular Conference. Vestibular disorders cause a…
Glut1 Virtual Community Gatherings: Teen and Adults with Glut1 Deficiency The Overcomers September 13th, 2020 The Glut1 Deficiency Foundation is not letting the coronavirus/COVID-19 pandemic stop them from continuing in…
The GRIN Virtual Conference September 12, 2020 Don't miss the CureGRIN Foundation's GRIN Virtual Conference. The event will be packed with valuable information for the GRIN disorder patient community. Participants…
Caroline Wozniacki has led an interesting life. Born in Denmark, she was immediately thrust into athleticism. Her father was a former football player (that's soccer for you Americans) and her…
Last month, biotechnology company BioMarin sought approval for an investigational gene therapy (valoctocogene roxaparvovec) for patients with hemophilia A. However, their approval was denied. Instead, the company was told that…
Cornell University and reVision Therapeutics have come together through a licensing agreement to develop a treatment for Stargardt disease, according to BioSpace. They will be working on and commercializing Cornell's…
According to a recent article in MedicalXpress, researchers in Japan led by Dr. Shinsuke Ishigaki and his team at Nagoya University, have identified new molecular details about tau’s activity. Alzheimer’s,…
According to a story from BioSpace, the radiopharmaceutical company Clarity Pharmaceuticals, Inc. has announced recently that its experimental diagnostic 64Cu-SARTATE™ has earned Rare Pediatric Disease designation from the US Food…
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