A few years ago, Dr. Thomas Weimbs, a researcher at the Weimbs Lab of UC Santa Barbara, performed a study on mice models of polycystic kidney disease (PKD). During…
In a recent article, English entertainer Tommy Steele has revealed what it was like being diagnosed with porphyria at a young age. Porphyria Porphyria is a group of disorders…
Recently, the International Society on Thrombosis and Haemostasis (ISTH) held its 2022 Congress from July 9th to 13th. During the conference, researchers and other stakeholders discussed research, care, and…
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…
CBC News Canada carried the story of a 23-year-old patient, Morgan Buyaki of Saskatchewan, Canada, who was diagnosed eighteen months ago with eosinophilic granulomatosis with polyangiitis (EGPA). Morgan’s mother…
Primary progressive aphasia (PPA) is a rare neurological disorder and form of frontotemporal dementia which affects one’s ability to communicate. In many cases, people develop PPA prior to turning…
Earlier this year, the European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) held its International Congress. During the Congress, researchers discussed the latest in research and scientific innovation within…
Having lived for over 20 years with chronic pain and persistent involuntary muscle contractions from dystonia, a neurological movement disorder, I have experienced a lot of suffering. If you have read…
Currently, there are few therapeutic options for people who become infected with the Nipah virus. Rather, treatment is symptomatic. However, that may soon be changing. According to the European…
A recent article on Yahoo Finance, reports that Alnylam Pharmaceutical Inc.’s RNAi therapeutic, Amvuttra, has been approved by the FDA to treat polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis. Amvuttra (Vutrisiran)…
In a recent article at Star Tribune, the Mayo Clinic's Dr. Julio Sartori Valinotti discusses excessive sweating, or hyperhidrosis, a condition that affects arounds 15 million people in the United…
Michael Klim was traveling three years ago when he felt like something within his body was wrong. The Polish-born swimmer, who has won two gold medals for Australia while participating…
Early diagnosis and treatment is crucial for patients with primary hyperoxaluria type 1 (PH1). Unfortunately, due to a high prevalence of misdiagnosis, as well as poor therapeutic access in…
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…
One thing is sure: 29-year-old Aimee Read was always a fighter. At just two years old, Aimee was diagnosed with leukemia. After two years of chemotherapy and six months…
According to a recent announcement, Epilepsy Research UK has announced a funding collaboration with the Tuberous Sclerosis Association which will support the investigation of tuberous sclerosis complex (TSC)-associated epilepsy.…
According to a story from Globe Newswire, the rare disease company Albireo Pharma, Inc. has recently announced the release of new data from its phase 3 trials. These trials were…
Currently, ZYNLONTA (loncastuximab tesirine-lpyl) is approved for the treatment of individuals with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) for which two prior therapies had not worked.…
The most common form of leukemia in Western countries is chronic lymphocytic leukemia (CLL). The disease accounts for almost thirty percent of known leukemia cases. Approximately 30,000 Europeans were…
According to a story from AJMC, there are a number of factors in play when determining the path forward for the treatment of advanced melanoma. For this story, Dr. Omid…
In the United States, during the drug development and approval process, drug developers are able to request Fast Track designation. This process is designed to facilitate and expedite the…
Medical research and clinical trials are extremely important tools in learning more about specific diseases, as well as understanding and evaluating potential treatments. For example, the Phase 2 VIBRANT…
According to a story from Healio, a recent phase 2 clinical trial found that treatment with vinorelbine plus active supportive care offered greater progression-free survival than active supportive care on…
Those living with rare diseases or conditions often face difficulty in finding treatment options - or even spurring researchers to make headway within their disease states. To combat this and…
A recent article at Healio Rheumatology highlights a presentation given by Dr. Arthur Kavanaugh of the University of California to attendees at the 2022 Clinical Rheumatology Congress East. Dr. Kavanaugh…
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