Growth differentiation factor 15 (GDF15) is expressed in normal conditions but is overly expressed when cells are under stress. It has multiple roles in different diseases and has been used…
In late January 2021, Dynacure, a clinical-stage drug development company focused on rare diseases, announced that it was granted a patent (U.S. Patent No. US 10, 865, 414) for its…
What are neuromuscular junctions and how do they play a role in the development of Charcot-Marie-Tooth disease (CMT)? According to Charcot-Marie-Tooth News, researchers discovered that structural neuromuscular junction changes caused…
In the Bomadi and Burutu areas of the state of Delta, within Nigeria, the main source of water is the Forcados River. According to Brittanica, the Forcados flows through freshwater…
A new licensing and supply deal has been made between Mitsubishi Tanabe Pharma America Inc. (MTPA) and Aquestive Therapeutics concerning the commercialization of riluzole in the United States. Riluzole, being…
A team of German Scientists is offering hope for people who have sports, traffic, or war-related injuries of the spinal cord. According to a recent article in Neuroscience News, these…
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…
How much does our diet play a role in underlying health issues? In some cases, it might play an even larger part than we believe. As recently reported in Yale…
Every year since 1954, the final Sunday in January has been recognized as World Leprosy Day. The day was first established by Raoul Follereau, a French writer and philanthropist. The…
Motor symptoms are not the only effects of Parkinson's disease; many affected individuals experience issues with sleep, among other symptoms. In fact, a panel of experts recently came together to…
In a recent press release, clinical-stage biopharmaceutical company Abeona Therapeutics Inc. ("Abeona") shared that the company had a successful Type B meeting with the FDA. During the meeting, Abeona explained…
At the very start of the new year, researchers out of the Washington University in St. Louis shared some exciting medical research: the discovery of two gene variants, associated with…
Parkinson’s News Today recently featured an upbeat article describing the progress that researchers have made in developing a method of measuring alpha-synuclein’s (AS) progression that cuts discovery time in…
Kuur Therapeutics has recently released interim data on their CAR-NKT cell therapy platform, which was created for the treatment of hematological and solid malignancies. This data comes from two phase…
In a press release shared at UEG week Virtual 2020, researchers shared a new study conducted over nearly two decades which found that people who’d had asthma or an allergy…
Each year, January 24 marks International Alagille Syndrome Awareness Day. During this day, organizations across the globe raise awareness around Alagille Syndrome (ALGS), from offering educational resources to allowing patients…
According to Medical XPress, researchers out of the Queen Mary University of London have been making strides in the field of medical research, particularly around pancreatic cancer. After identifying a…
Written by Neil Smith I have a rare hormonal condition called Kallmann syndrome. Its main symptom is a failure to start or fully complete puberty. It is also associated with…
Written by Jennifer Peterson As a young adult, this journey was not something I ever imagined taking, and it certainly was not for the faint of heart. Being diagnosed with…
According to a story from Business Weekly, the biotechnology company Healx, from Cambridge, UK, and the US-based Mission: Cure coalition have announced a new partnership which aims to help develop…
There are many common elements in Alzheimer’s disease (AD), which is the most common type of dementia. It is now evident that there are varied spans of disease progression…
According to a press release from the rare liver disease company Albireo Pharma, Inc., the US Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for odevixibat.…
The National Institute for Health and Clinical Excellence (NICE), an agency under the UK's National Health Service (NHS), has recently rejected Zeposia. Zeposia is an oral, multiple sclerosis (MS) treatment…
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…
Although advances in lung cancer care were overshadowed this year by news about the pandemic, Dr. Jennifer Carlisle’s recent interview with Oncology gave details of spectacular advances in lung cancer…
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