Tarsius Pharmaceuticals has just announced positive results for their recent trial for uveitic glaucoma. This trial was called GADOT 20/20 and it was investigating a therapy called TRSo1. Ocular Diseases…
Last week, clinical stage biopharmaceutical company Neurotrope announced that the first patient was dosed in a Phase 2 clinical trial to test the safety, efficacy, and tolerability of bryostatin-1…
Grifols, a pharmaceutical company based out of Barcelona, Spain, has announced that their potential treatment for the novel coronavirus has entered a phase 3 trial, according to BioSpace. It is…
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…
According to a story from GlobeNewswire, the biopharmaceutical company X4 Pharmaceuticals, Inc. has just announced that the US Food and Drug Administration (FDA) has granted its investigational product candidate mavorixafor…
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…
According to a story from Pharma Advancement, Bristol Myers Squibb recently announced encouraging findings from a phase 3 clinical trial. This study was testing its drug ozanimod (marketed as Zeposia)…
In the past, there existed no approved treatments for alkaptonuria (AKU), a rare genetic disorder characterized by homogentisate dioxygenase deficiency. However, according to Medical XPress, this recently changed as British…
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…
In conditions like multiple sclerosis or neuromyelitis optica, disease progression is spurred by the destruction of myelin, the protective coating around nerve cells. However, new research suggests that N-acetylglucosamine, a…
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…
For years, clinical-stage biopharmaceutical company Fulcrum Therapeutics ("Fulcrum") has worked to develop therapies for patients with rare genetic disorders and diseases. Somewhat recently, their focus was on treating beta thalassemia…
If you're like me, you might be surprised that COVID-19 is not only still deeply ingrained in our world, but it seems to continue growing. As of the afternoon of…
At the start of October, Swedish-based IRLAB announced that the FDA approved an investigational new drug application (IND) for their therapeutic candidate mesdopetam (IRL790). This therapy is designed to treat…
According to a press release, biopharmaceutical company Apellis Pharmaceuticals ("Apellis") recently launched registration programs to explore pegcetacoplan for patients with C3 glomerulopathy (C3G), amyotrophic lateral sclerosis (ALS), and immune…
According to a story from Muscular Dystrophy News Today, a gene therapy in development by the pharma company Pfizer, Inc. was recently given Fast Track designation from the US Food…
The FDA has recently granted the Breakthrough Therapy designation to FARXIGA, a chronic kidney disease treatment created by AstraZeneca. This designation will allow for a quicker development, which will hopefully…
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