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…
In a previous article, I talked about the importance of patient-led advocacy groups and how they can positively impact how a rare diagnosis is received and understood by patients. Do…
According to a recent news release, the FDA cleared an Investigational New Drug (IND) application for VIP152, a potential therapeutic option for patients with Richter syndrome and relapsed/refractory chronic…
Alarming details of Katy Grainger’s sepsis infection were covered in the April 2021 issue of Women’s Magazine. The interview took place over two years after Katy lost both feet and…
According to HCP Live, a new study may have discovered a connection between severe pediatric atopic dermatitis and learning disabilities. Researchers, spearheaded by Dr. Joy Wan, MD, MSCE from the…
Scholarships are being provided to rare disease patients, thanks to the EveryLife Foundation for Rare Diseases. The scholarships are each worth $5,000 and are intended for those seeking an education…
According to a story from globenewswire.com, the clinical company Prothena Corporation plc recently presented encouraging results from its phase 1 clinical trial. This trial was evaluating its investigational product candidate…
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…
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