Guadalupe Hayes-Mota has worked for Biogen, Ultragenyx, Amgen, and GSK. He was the prior UCLA Health Director. Additionally, he is a member of the Massachusetts Rare Disease Advisory Council. He…
Clinical trials can be beneficial for finding and evaluating new treatment options. Recently, biopharmaceutical company AstraZeneca shared how ALXN1840, a potential treatment for patients with Wilson disease, reached its primary…
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…
A recent study investigated the experiences of patients with chronic hypoparathyroidism in pregnancy. The study ultimately found that these patients typically had low risk pregnancies and normal outcomes. However, they…
A recent study has found that when eosinophilic esophagitis is not treated, it can become progressive. These findings demonstrate how important it is for patients to be regularly monitored and…
Patient Worthy was pleased to serve on the advisory committee for the development of this new resource. Caring for a child with a rare disease can be scary and confusing;…
The National Organization for Rare Disorders (NORD) has been a leading patient advocacy organization since the 1980s, helping advance the identification, treatment, and cure of rare disorders. In October 2021,…
Ashley Monroe is an extremely talented singer-songwriter who hails from Tennessee. Over the years, Ashley has grown her music career alone and as part of the Pistol Annies. But now…
When it comes to pharmaceuticals, licensed territory helps ensure that drugs within a certain territory are offered to patients. Exclusive licenses sometimes mean that pharmaceuticals are only available in specific…
For rare diseases in which no current treatment options are available, such as GM1 gangliosidosis, there is an urgent need to find therapeutics and improve patient outcomes. Gene therapy company…
Meghan Thompson, a doctor and 3rd year fellow at Memorial Sloan Kettering Cancer Center has recently explained all of the emerging treatments using BTK inhibitors to treat CLL, chronic lymphocytic…
Paroxysmal nocturnal hemoglobinuria (PNH) occurs from a mutated PIG-Agene. This causes blood cells to be produced which don't have surface proteins. As such, the cells aren't protected adequately from the complement…
Rare disease patients often face more obstacles and receive less aid than those impacted by more common conditions. There are many reasons for this inequity, but more research must be…
Each year, the National Kidney Foundation (NKF) holds a Kidney Walk to help provide support and assistance to those with chronic kidney disease (CKD) and other kidney-related conditions. Due to…
Merck has just received FDA approval for their recently acquired drug Welireg. This HIF-2α blocker is indicated for von Hippel-Lindau disease (VHL). This rare disease gives affected individuals a higher…
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…
Have you ever wanted to be part of medical research? Well, now you might have the chance. According to Fox2Now, researchers from Saint Louis University - and, in particular, the…
Nearly all cases of Wilms tumors, a rare form of pediatric kidney cancer, are diagnosed before a child turns 10 years old. When Maddie Newburn was first diagnosed, she was…
I think everybody would agree that stress is a challenge in their lives. Personally, I think we should take on the belief that no stress exists and that it is…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat patients with rare conditions, defined as affecting fewer than 200,000 Americans. As incentives, drug…
AstraZeneca is ending a phase 3 trial of Ultomiris, which was being evaluated as a treatment for amyotrophic lateral sclerosis (ALS). The pharmaceutical company acquired the C5 inhibitor after buying…
When Karter was first born 14 months ago, his mother Breonna was overjoyed. But she quickly realized a series of upcoming obstacles when, at birth, Karter was diagnosed with Alagille…
Normally, the standard FDA review process for potential drugs or biologics takes around 10 months to complete. However, when a drug receives Priority Review status, it means that the FDA…
According to a recent press release from Pfizer and biotechnology company Vivet Therapeutics, VTX-801, a gene therapy candidate for patients with Wilson disease, recently received Fast Track designation from the…
When organizations partner together to work towards improving patient outcomes, it can also help to increase the understanding and spur research of certain conditions. According to Healio, a recent…
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