The very first patient has just been dosed in Pfizer's Phase 3 clinical trial for Duchenne muscular dystrophy (DMD). The trial is called CIFFREO. This patient received the therapy at a…
Toxoplasmosis is transmitted by Toxoplasma gondii, the protozoan parasite which is known to be among the most common parasites worldwide. The infection occurs from contaminated meat, cat feces that…
Illumina has just formed a new partnership with Emedgene. Emedgene is an AI company which works to automatically interpret and collect data for rare genetic diseases. This data will be…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
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…
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…
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…
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…
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…
Sorafenib, a tyrosine kinase inhibitor, has been unbeatable for treating cancer of the liver for the past thirteen years. An article by the Cleveland Clinic stated that during that time,…
Medical professionals have known for a long time that peripheral nerve cell damage plays a major role in Charcot-Marie-Tooth (CMT) disease. A recent study published in Human Molecular Genetics has suggested that…
Over the past decade, scientists have made many life-saving advances in all areas of medicine. They have created molecular agents to target neurodegenerative diseases. But they were stopped right there…
Welcome to study of the week, a new series from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is…
According to a story from newswise.com, a team of researchers from the University of New Hampshire may have discovered the next new treatment for a rare form of blood cancer…
Parent Project Muscular Dystrophy's (PPMD) goal is to end Duchenne muscular dystrophy (DMD), and they've just made another step in the right direction. They have recently announced the launch of…
Researchers at Johns Hopkins School of Medicine noted that osteonecrosis (ON) is one of the more serious complications of systemic lupus erythematosus (SLE). According to a recent article in…
Patient input is extremely important when evaluating treatments for rare diseases; Charcot-Marie-Tooth disease (CMT) is no exception. In order to understand the impact of different CMT therapies, researchers from the…
Neuroscience News recently carried an article heralding reports of researchers' finding that upwards of twenty percent of lethal glioblastoma brain cancers may be treatable by newly developed drugs. Currently, the…
Translate Bio has been moving forward with the development of their new drug candidate, MRT5005. This mRNA therapy was created to address the underlying cause of cystic fibrosis (CF), and…
According to a study on hindawi.com, the presence of abnormally elevated levels of serum IgG4 was correlated with a greater likelihood of patients living with autoimmune hepatitis to present with…
Diagnosing Rare Diseases Diagnosing rare diseases is historically difficult. Physicians are taught to think about horses instead of zebras because horses are abundantly more common. For instance, if you're tired…
Takeda has just announced that their application to the the FDA for their eosinophilic esophagitis (EoE) investigative therapy has received Priority Review. This therapy is called budesonide oral suspension or…
According to an article in BioSpace, Dyne Therapeutics has recently announced data from preclinical trials of its FORCE platform. This program is aimed at creating treatments for myotonic dystrophy type…
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