Currently, there are a number of treatment options for patients with follicular lymphoma. But for patients whose condition has relapsed despite prior treatments, there are very limited therapeutic options. After…
Jaguar Health and Napo EU S.p.A have just announced that crofelemer, an investigative therapy for short bowel syndrome (SBS) has been granted Orphan Drug Designation by the EMA. Thanks to…
Diagnosing a rare disease early is important, as it allows for prompt intervention and treatment. Recently, researchers took large steps forward in the diagnostics of pulmonary arterial hypertension (PAH) in…
Rare disease patients know what it's like to go without a diagnosis despite enduring symptoms. Doctors often don't recognize the disorders for a number of reasons, whether that's the similarity…
A recent study published in Advances in Rheumatology has uncovered 2 potential biomarkers which could be used to predict which lupus nephritis (LN) patients are at the highest risk of severe renal…
Researchers from the Perelman School of Medicine at the University of Pennsylvania recently explored the underlying biological cause and mechanisms behind fibrodysplasia ossificans progressiva (FOP). In the past, researchers from…
A recent article showed how the parents of a little girl with the rare blood disorder Von Willebrand disease are spreading awareness about the importance of donating blood. Von Willebrand…
A study recently published in the American Journal of Transplantation evaluated whether or not transplanting the kidney of a pig could be a viable and safe options for human patients. This study…
Just about two years ago, Jane Warrimer began experiencing abnormal menstruation. Her period was no longer regular; her bleeding lasted for weeks at a time. After pursuing an IUD, Jane…
Have you ever heard of an astrocyte? This star-shaped cell is found throughout the nervous system. Normally, healthy astrocytes protect motor neurons from damage or degeneration. But according to Medical…
Tim Smith is the President of Many Faces of Moebius Syndrome (MFOMS), a nonprofit spreading awareness of the rare condition Moebius syndrome. This condition is characterized by the underdevelopment of…
According to a press release from the pharmaceutical company Bristol Myers Squibb, the company's CAR T-Cell therapy idecabtagene vicleucel (marketed as Abecma) has been approved by the Japanese Ministry of…
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…
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