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 FDA has just announced its accelerated approval of the very first therapy which can reduce proteinuria (high levels of protein in the urine) in patients diagnosed with primary immunoglobulin…
Victoria Gray’s recent interview with NPR confirmed that her genetically modified blood cells have truly transformed her life. Victoria volunteered in the first-ever attempt in the U.S. to use CRISPR,…
In a news release from January 4, 2022, biopharmaceutical company SpliSense shared that their drug candidate SPL84-23-1 for cystic fibrosis (CF) earned Orphan Drug designation in both the United States…
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 a…
In Part 1 of "With Mais and Mal," Patient Worthy spoke with Mallory Cyr about microvillus inclusion disease (MID), Mallory's story, and the process of becoming an advocate. Today, we…
A study conducted by Rayyan Zafar and his colleagues from the Imperial College London was recently published in BMJ Paediatrics Open. This investigation evaluated the efficacy of whole-plant cannabis as a treatment for…
According to a recent article, a low carbohydrate intervention used to reduce type 2 diabetes and for prediabetes regression, may be a treatment option for patients with nonalcoholic fatty liver…
Most people have heard of Viagra, and they know it as a treatment for erectile dysfunction (ED). Fewer people know it as Revatio, which is indicated for pulmonary arterial hypertension…
According to a recent article, a new study shows a daily oral dose of Belzutifan may keep patients diagnosed with von Hippel-Lindau disease from needing surgery by shrinking tumors. Von…
According to a recent article, a new study from Boston Children’s Hospital shows that a first-of-its-kind gene therapy technique has succeeded in reversing severe hearing loss from a mutation to…
Givlaari, a treatment for acute hepatic porphyria (AHP), was first approved in Europe back in March of 2020. However, approval doesn't mean immediate access unfortunately. It's been nearly two years…
What does someone with a rare disease look like? With over 7,000 rare diseases in the world, affecting an estimated 300 million people, life with a rare disease (and people…
Positive results have been announced from a Phase 2 trial of olutasidenib, an investigational selective mIDH1 inhibitor used in combination with azacitidine for the treatment of the mIDH1 form of…
The FDA has recently granted Accelerated Approval to Tarpeyo, also called budesonide, to reduce proteinuria in patients with IgA nephropathy (IgAN). Tarpeyo is the first and currently the only treatment…
After several clinical setbacks, Vertex Pharmaceuticals found promising results in its study of a drug to treat a genetic kidney disease called focal segmental glomerulosclerosis (FSGS). As reported recently by…
Dr. Joshua Sabari, a professor at New York University's Department of Medicine, has recently spoken about the different treatment options for ALK-positive non-small cell lung cancer (NSCLC). Specifically, he discussed…
Cannabidiol (CBD) has been explored as a potential therapy for a variety of different conditions, ranging from various forms of epilepsy to dystonia to Parkinson's disease. Now, researchers are questioning…
A recent article published in Vascular News highlights the importance of plasma desmosine, an elastin component, that provides elasticity for tissue. A breakdown of elastin leads to aortic dilatation and…
Non-clear cell renal cell carcinoma (nccRCC) has recently been found to have a very strong connection with various subtypes of proliferative disease. Further, this condition was found to have a…
Healio recently published results of the CheckMate 649 study confirming the benefits of combining nivolumab and chemotherapy in treating newly diagnosed patients with gastroesophageal junction cancer and advanced gastric and…
Have you ever heard of the cytokines interleukin-6 (IL-6) or TNF? Normally, these cytokines, excreted by the immune system, may influence autoimmunity or immune responses to foreign invaders. For…
Acknowledgment: This story is sponsored by CymaBay Therapeutics and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing relevant, vetted and valuable information…
The FDA grants the Orphan Drug designation to treatments indicated for rare diseases, which are defined as affecting less than 200,000 people in the U.S. This designation is meant to…
Pancreatic cancer can present numerous problems for medical professionals. To start, this cancer can be difficult to detect and diagnose in early stages. By the time pancreatic cancer progresses,…
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