According to a recent article from BusinessWire, the European Commission (EC) has approved KEYTRUDA as a monotherapy for adult patients with renal cell carcinoma (RCC). Specifically, this approval is indicated…
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…
The World Health Organization has recently declared that they will beat meningitis by 2030 by assisting countries in mitigating its spread and minimizing the effects of the disease itself. They…
At the end of last year, the FDA warned people with hyperkalemia to monitor their diets when it comes to potassium intake. According to an article from the Taipei Times,…
For the last seven years, the Global Genes Rare Compassion Program has been providing medical students with an opportunity to bond with patients and families who have been diagnosed with…
Our parents can be some of our strongest supporters, and Hawken Miller knows this firsthand. He recently published an article in Muscular Dystrophy News Today detailing the love and support…
Each year, approximately 1 in every 1 million infants is diagnosed with pediatric myelodysplastic syndromes (MDS), a group of rare disorders characterized by the abnormal or deficient formation of blood…
Vision loss is a symptom associated with a number of disorders, one of which is Usher syndrome. Medical professionals have spent years trying to fully comprehend the underlying mechanisms of…
U.S. News carried an article reporting that in a recent study, researchers have identified minute amounts of cancer (organoids) in fallopian tube tissue. The significance of the discovery is that…
Because there are many obstacles that stand in the way of developing drugs for rare diseases, the FDA grants a number of designations to incentivize companies to create these treatments.…
Professor Miranda Orr of Winston Salem’s School of Medicine in North Carolina leads a team of investigators who have detected senescent cells in patients with Alzheimer’s disease. These cells, found…
According to a recent press release from Dyne Therapeutics, the FDA has placed a clinical hold on their Investigational New Drug (IND) application for DYNE-251. Until this hold is lifted,…
Seven years ago, at just 15 years old, Lauren Blake was diagnosed with gastroparesis. This condition makes it difficult for Lauren to keep down her meals or, really, to eat…
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 World Health Organization (the WHO) announced World Leprosy Day which this year will be celebrated on January 30th. The theme of the celebration is “United for Dignity.” It…
For years, there have been few – or no – therapeutic options in Canada for those with fibrodysplasia ossificans progressiva (FOP), a rare genetic connective tissue disorder. However, this has…
Julia Lefelar, a resident of Gaithersburg, MD, was telling a friend about her years of seeing different doctors trying to find the cause of her illness. After a while, her…
Orphan Drug designation is granted to drugs or biologics intending to treat, prevent, or diagnose rare diseases or conditions. For the purpose of the designation, “rare” is considered any condition…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is…
According to a recent article from Hematology Advisor, a study shows patients diagnosed with particular subtypes of diffuse large B-cell lymphoma (DLBCL) respond better to ibrutinib with R-CHOP compared to…
The Critical Path Institute (C-Path), formed in 2005, is a nonprofit, independent (public/private) partnership. The Institute evolved from the FDA’s 2004 White Paper known as the Critical Path Report and…
In a recent news release, enzyme engineering company Codexis, Inc. shared that its product CDX-6512 received both Orphan Drug and Rare Pediatric Disease designations from the FDA. Both designations were…
A recent article published in Physician's Weekly highlighted a study that compared four different treatment regimens for lupus nephritis. The options include low-dose cyclophosphamide (LDCyC), high-dose cyclophosphamide (HDCyC), rituximab, and…
According to a recent article, a recent study shows evidence that anti-Semaphorin 3B antibodies are pathogenic and therefore patients diagnosed with membranous nephropathy should be monitored after a kidney transplant.…
According to a recent article, a new study has developed a novel index that differentiates eyes with subclinical keratoconus and normal eyes. Keratoconus Keratoconus is a disorder affecting the cornea…
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