Normally, metformin is used to help control blood sugar in patients with type 2 diabetes. However, new research suggests that the treatment option could also be beneficial for those with…
According to Medical Xpress, a team of researchers from The Wenner-Gren Institute at Stockholm University and Karolinska Institutet, Karolinska University Hospital has conducted a study that investigates neuronal death in…
In a recent interview with CNN, Dan Schoenthal, a retired 56-year-old police officer of Great Valley, New York, explained that in 2015 he received a diagnosis of Parkinson’s disease.…
Dan Baker, a 56 year old former anesthesiologist, knew from childhood that something peculiar and unpleasant was going on with his body. Unfortunately, it would take decades before he found…
Prime editing, a new entry in gene-editing technology, is said to be an extension of the “genetic toolbox.” According to a recent article in Science Daily, prime editing designs…
It is now commonplace for patients to find that a drug such as prednisone, usually prescribed for relief of allergic and rheumatic symptoms, is also prescribed to treat leukemia. According…
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…
Gene editing, CRISPR specifically, has become much more well-known and heavily used in the recent past. Advances in this field have allowed medical professionals to treat conditions that had seen…
According to FirstWord Pharma, patients with paroxysmal nocturnal hemoglobinuria (PNH) in the United Kingdom (UK) may soon have a more effective and efficient, and less invasive, treatment option. Currently, the…
In 2009, the FDA approved Tyvaso (treprostinil) Inhalation Solution to reduce symptoms associated with Group 1 pulmonary hypertension (PH), also known as pulmonary arterial hypertension (PAH). Beyond symptom reduction, the…
For small patient groups, creating momentum towards rare disease solutions often seems futile. To tackle such expensive and extensive endeavors, rare communities find strength and hope by putting their minds…
Researchers have been able to identify similarities in outcomes between colorectal liver metastases that cannot be completely removed by surgery (unresectable) and patients with the KRAS mutation. Surgical resection…
According to Healio, an open-label study of oral octreotide has reported that the treatment is able to maintain its response in acromegaly patients without additional safety signals. This data is…
C. difficile is a bacterial infection that impacts the gastrointestinal system. It can lead to weight loss, diarrhea, dehydration, and even the loss of blood platelets. Megan Newman has had…
Lyme disease is a vector-borne disease spread through ticks. It is most common in the Northeast, North-Central, and mid-Atlantic portions of the country, but is notably absent in the Southeast.…
Growing up, I was always told that oral health related directly to regular health. But now, a study from researchers at the NYU College of Dentistry and Weill Cornell Medicine…
Sawyer Burch is a four-year-old boy from the Nashville, Tennessee area who was born with trifunctional protein deficiency, a rare disorder that takes the body's ability to utilize certain types…
Passage Bio has recently announced that Orphan Drug Designation has been granted by the European Commission for PBKR03, a gene therapy for Krabbe Disease. This condition has yet to have…
In mid-April, Kantar Health and the International Gaucher Alliance (IGA) shared the launch of a rare disease registry developed by the partnership. The registry, known as GARDIAN, will explore Gaucher…
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…
Earlier this year, WP1066, a p-STAT3 inhibitor, received three separate Rare Pediatric Disease designations for the treatment of medulloblastoma, atypical teratoid rhabdoid tumor, and diffuse intrinsic pontine glioma. Now,…
Clinical trials can be beneficial in many ways. For one, these trials show researchers whether a treatment’s impact is durable and sustained. Determining this often requires some follow-up period. According…
Inbrain Neuroelectronics has announced that they have raised 16.8 million dollars which will support clinical testing of a new device for Parkinson's disease as well as epilepsy. This is a…
Whitney Dafoe was an aspiring photographer who backpacked across the world. However, he always would return from his trips extremely exhausted. He would feel weak and he didn't know why.…
According to Drug Target Reviews, there may be an emerging vaccination effective against preventing Zika virus infection. Though many cases are mild, the infection can cause severe birth defects and…
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