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,…
According to a recent press release from uniQure, the first round of observations from a trial investigating AMT-130 for Huntington's disease (HD) have been released. As of now, four patients…
January is Cervical Health Awareness Month! So, why not use the momentum and goal-making spirit of New Year's Resolutions to make sure you get screened for cervical cancer and schedule…
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