There is a need for more research into Leigh syndrome, as there are currently no FDA-approved treatments, and the patient population faces an unmet need. However, in order for there…
Scientists at London’s University College and a team of scientists at Stanford University simultaneously made the same discovery about amyotrophic lateral sclerosis (ALS): the genetic variant in the protein UNC13A…
Have you ever heard of a biomarker? In short, a biomarker, or biological marker, is some sort of objective measure which can tell you something about your health or health…
The European Commission (EC) has recently granted its Orphan Drug designation to AR101, a PKCβ inhibitor for the treatment of Ehlers-Danlos syndrome (EDS). This decision was based off of a…
When Rachel Matthews birthed her twin boys, Aiden and Gavin, she was ecstatic. But just a day or two after Rachel had returned home with her boys, she received a…
There are three subsets of Von Willebrand disease (vWD), a genetic bleeding disorder. Type 3 is considered to be the most severe form of vWD, culminating in intense or severe…
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…
Patients with advanced kidney cancer have a new option in a combination therapy that will afford them a longer and better life. Kidney cancer can be hard to cure, but…
February 28, 2022 is celebrated annually as Rare Disease Day. This is a major event for the rare disease patient community that helps spread awareness and shine a light on…
According to a recent article, Trialbee and ERGOMED have partnered to become the platform for patient recruitment for their new Rare Disease Innovation Center. Trialbee Trialbee is the leading platform…
Eosinophilic-associated disorders are a group of conditions characterized by heightened levels of eosinophils, which are a type of white blood cell within the immune system. One of these disorders is…
The FDA has recently announced that they have put a clinical trial for relapsed or refractory T-cell lymphoma patients and cutaneous T-cell lymphoma patients on hold. This trial was a…
If you follow Major League Baseball (MLB), you’ve probably been inundated with news regarding the current lockout – and concerns over whether the season will begin on time. However, if…
March 3, 2022 is being recognized at International TAPS Day, a time to spread awareness among the general public and the medical field about twin anemia polycythemia sequence (TAPS) a…
According to a recent article from MedicineNet, scientists are editing the genomes of ticks to help prevent tick-borne diseases such as Lyme disease. Lyme Disease Lyme disease is a vector-borne…
According to an article from Healio, studies have proven that budesonide oral suspension (BOS) is a viable treatment option for eosinophilic esophagitis (EoE). Research demonstrated that BOS outperformed placebo in…
According to a recent article, researchers have found that adding durvalumab to chemotherapy treatments results in prolonged survival rates in patients with advanced biliary tract cancer. Biliary Tract Cancer Biliary…
The FDA has just announced that they are reevaluating their approvals for Ukoniq. This reevaluation comes after study findings which demonstrated there may be an increased risk of fatality for…
How effective is UPLIZNA on treating attacks associated with neuromyelitis optica spectrum disorder (NMOSD)? According to a news release from biopharmaceutical company Horizon Therapeutics plc ("Horizon"), an analysis highlighted that…
Prior to February 2022, there were no approved treatments for those with cold agglutinin disease (CAD). However, that changed on February 4th, 2022, when the FDA announced that it had…
A new, joint research pact between Illumina and the National Cancer Center Japan aims to shed light onto the clinical information and genomics of nasopharyngeal carcinoma, a rare cancer. Together,…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions. A “rare” condition is one affecting under…
Nicole Burns’ great grandfather left her his house and a portion of his 100 acres. According to a recent article in STAT News, Nicole also inherited her great grandfather’s heart…
BRUKINSA (zanubrutinib) is a prescription medication which can be used to treat Waldenstrom macroglobulinemia (WM), mantle cell lymphoma, and marginal cell lymphoma. So far, the treatment has been approved in…
Historically, both osteosarcoma and neuroblastoma have been difficult to treat. Therefore, new treatment options are needed for these cancers, which predominantly affect children and teenagers. According to Medical XPress, a…
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