According to a story from GlobeNewswire, the medical device company SoniVie announced recently that the US Food and Drug Administration (FDA) has granted approval for its Investigational Device Exemption (IDE).…
The United States FDA has just announced that they will be providing 6 new grants, which are worth 16 million dollars for rare disease research. These grants will last a…
On October 6, 2020, molecular engineering company Scribe Therapeutics announced an exciting partnership. Originally founded by molecular engineers and CRISPR inventors, Scribe Therapeutics seeks to develop in vivo genetic medicine to treat…
Fetal alcohol spectrum disorder (FASD) is a spectrum of disorders that can impact decision making and make individuals unable to understand the consequences of their actions. Because of this, it…
EB Research Partnership (EBRP) is based out of New York, United States. EB Research Foundation (EBRF) is an organization based out of Australia with the same goals. The two organizations have…
In December 2019, the FDA approved Ervebo, the first-ever Ebola vaccine. However, despite preventative measures, there still was never any actually approved treatment options - until now. Recently, the FDA…
Over this past week, the Harrington Discovery Institute announced five recipients of its introductory Harrington UK Rare Disease Scholar Award competition. The Institute, which is involved with Cleveland's University Hospitals,…
Dr. Justin Hopkin's son Garrett was born happy and healthy, but when he was around four to six months old, his parents began to notice some problems. Garrett was having…
J.G. Jones is a renowned comic book artist, illustrating covers of beloved superhero novels like Batman and Robin, Doc Savage, and Frankenstein. He has probably worked for all of the…
Happy Friday! October is going by quickly, but if you need something else to help pass the time, why not give our podcast a listen? The two newest episodes of,…
According to a story from GlobeNewswire, the biopharmaceutical company Immunomedics, Inc., has recently announced that its medication sacituzumab govitecan-hziy (marketed as Trodelvy) has recently earned Orphan Drug designation from the…
Researchers have just uncovered 30 new genes that may cause Charcot-Marie-Tooth Disease (CMT). These genes were uncovered using computer analyses of biological data, a process called bioinformatics. This study was published…
According to a recent article in BioSpace, young children and infants who have been diagnosed with primary hyperoxaluria type 1 (PH1) are often faced with the use of gastrostomy…
Patient Worthy has recently begun a partnership with the Glanzmann's Research Foundation, a nonprofit patient organization dedicated to spreading awareness about Glanzmann's thrombasthenia and finding a cure. We spoke with…
The receptors in the human epidermal growth factor receptor 2 (the HER2 ) gene are active participants in determining whether healthy breasts can repair themselves. According to a recent article…
Living with an invisible illness can be incredibly difficult. Those around you do not understand the struggle, and they can even discount your illness. Women's Health talked to three women…
CIDRAP recently featured JAMA NetworkOpen's online reports of two timely research letters. The letters outlined the complications that may arise for aortic stenosis patients who have had to delay transaortic valve…
Researchers have recently conducted a study using mouse models of Charcot-Marie-Tooth (CMT) disease, titled "CMTM6 Expressed on the Adaxonal Scwann Cells Surface Restricts Axonal Diameters in Peripheral Nerves." It was…
Waking up to the news has one encouraging note, another new day and another new drug is being tested. According to an article in Biospace, the news from Sorrento Therapeutics…
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…
As published in a press release by the Hereditary Neuropathy Foundation, a project funded by the National Institutes of Health (NIH), researchers at the University of Rochester, the University of Pennsylvania, and…
The FDA has recently granted the Fast Track designation to AGLE-102, a treatment for dystrophic epidermolysis bullosa. This designation will allow for the treatment to reach patients at a quicker…
When it comes to rare disease research and drug development, there is one crucial part of the equation: patient input. After all, how can companies ensure that they are meeting…
Near the end of September, Hansa Biopharma ("Hansa") announced promising data from a Phase 2 clinical trial on imlifidase. This therapy is designed to treat patients with severe anti-GBM antibody…
As reported recently in SciTech Daily, for years scientists have recognized red blood cells (RBC) as a vehicle to deliver drugs within the human body. They circulate for days and…
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