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…
A rare disease patient's medical odyssey often begins with a search. Rhisa Perera wrote a narrative in lymedisease.org about her long journey navigating the medical world to find answers and…
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…
This past week, Target RWE announced data from its TARGET-NASH study, designed to better understand differences in health outcomes for patients with nonalcoholic fatty liver disease (NAFLD). According to their…
According to a story from BioSpace, the drug company Plus Therapeutics has recently announced that its investigational therapy Rhenium NanoLiposomes (RNL™) has earned Orphan Drug designation from the US Food…
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…
Happy Friday! Sept. 13-19 is Balance Awareness Week. It's intended to bring attention to balance-related vestibular disorders such as Meniere’s disease and vestibular migraine. Looking for ways to help out?…
According to a story from BioSpace, the biopharmaceutical company POXEL SA has recently announced that it has commenced with a phase 2 dose ranging clinical trial which will test the…
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 Buzzfeed News, the coronavirus/COVID-19 pandemic continues to present new surprises and challenges to the medicine and science communities. In a recent interview, Wayne Sebastianelli, who…
Bethany Buettner had started coughing...a lot. She had recently faced pneumonia, but after a course of antibiotics she appeared to have recovered...except for her persistent cough. It wasn't long before…
According to a story from Pharmaceutical Business Review, the pharmaceutical company Bayer has recently announced encouraging findings from its phase 4 clinical study. This trial was testing the drug riociguat…
Between August 29 and September 1, researchers joined in at ESC 2020 - The Digital Experience, developed and organized by the European Society of Cardiology (ESC). During this online conference,…
To determine the safety and efficacy of experimental gene therapy ST-920, researchers first examined the impact of the drug on mouse models of Fabry disease. As reported in Fabry Disease…
Early this week, biopharmaceutical company Avidity Biosciences announced its inclusion into a collaborative natural history study on myotonic dystrophy type 1 (DM1). The study, END-DM1, is run by the Myotonic…
As reported in Biospace, the life sciences company Revive Therapeutics Ltd. has announced their intention to join a Clinical Trial Agreement (CTA) with the Board of Regents of the University of…
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…
To determine disease progression and severity, researchers often use biomarkers, a measurable substance that indicates disease state. According to Myasthenia Gravis News, a new study shows that acetylcholine receptor (AChR)…
Ionis Pharmaceuticals has just released their findings from a study investigating the effect of IONIS-PKK-LRx and IONIS-PKKRx in hereditary angioedema (HAE) patients with different forms of the disease. These therapies work to reduce…
A new study published in the journal Rheumatology and Therapy examined the effect of baricitinib on Rheumatoid Arthritis (RA) patients who have had an inadequate to their methotrexate therapy. Methotrexate and Baricitinib…
Researchers in Canada have just released positive results from their heterozygous familial hypercholesterolemia (HeFH) clinical trial. This investigation was examining the effect of Repatha on teenagers with the condition. The trial…
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