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…
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…
A new genetic test for keratoconus as well as other types of corneal dystrophy was released late last year. The test was created by Avellino and can identify biomarkers of disease. It also looks…
Rocket Pharmaceuticals has recently dosed the first patient in their trial of RP-A501, a treatment for Danon disease. According to BioSpace, this trial will test the safety, tolerability, and efficacy…
Research was presented at The Digital International Liver Congress that proved nidufexor is a safe and viable treatment for nonalcoholic steatohepatitis (NASH), according to Healio. It was shown to reduce body…
The CureGRIN Foundation has recently announced that its annual conference will be held virtually this year as a result of the ongoing coronavirus/COVID-19 pandemic. This year's event will be held…
According to Charcot-Marie-Tooth News, the EveryLife Foundation has began a scholarship fund to help rare disease patients in the United States. Living with a rare disease often brings challenges, many…
Elliot Preddie, age 12, is ready to get back to his favorite sport, football. He had to put off playing due to sickle cell disease (SCD). Elliot substituted taekwondo and…
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