Policy regarding many topics varies throughout America as one moves from state to state, and rare disease policy is no different. Because of this, getting a holistic view of where…
The FDA has recently approved a treatment called Pyrukynd as a therapeutic option for hemolytic anemia disorder sparked by pyruvate kinase deficiency. Hemolytic anemia is a condition where red blood…
According to a recent article, a team at the Heart Institute is working on creating a guide to help cardiologists decide what treatment is best for patients diagnosed with anomalous…
A recent study examined the impact of age on clinical outcomes for patients diagnosed with chronic lymphocytic leukemia (CLL). This study was published in the journal Blood Advances. All patients in…
The question plaguing the geneticists about the Burns family was: how have they survived? In 2016, several geneticists theorized that certain people may have a good-gene bad-gene scenario taking place…
According to a recent article, there are a few warning signs of nasopharyngeal cancer that affect the lower half of your face that you might not be aware of. Cancer…
Written By Joslyn Crowe Caring for someone with a rare disease, especially a child or a family member, can be a difficult and emotional experience. Many people become a caregiver…
Silmitasertib, an investigative therapy for biliary tract cancer (BTC), has been awarded Orphan Drug Designation by the FDA. Researchers and the regulatory agency are hopeful that this therapy could become…
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…
According to a recent article, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) is now recommending changing the marketing authorization for avapritinib, which is used…
The 300 million people living with rare diseases seldom receive appropriate medical treatment. Unlike common chronic diseases for which there are established and constantly evolving treatments and interventions, those with…
Judy was diagnosed at age 45 with the rare disease IgA nephropathy in 2019. The diagnosis changed her life forever. IgA nephropathy is difficult to diagnose until it has progressed,…
A popular book by Dr. Seuss that is often given to graduates is Oh The Places You'll Go. Now, suppose Dr. Seuss had entered the rare disease world. Perhaps he…
Written by Matt Horsnell, Community Manager, TREND Community TREND Community is a leader in evidence acceleration through social data exploration for rare and chronic illnesses. Patient Worthy is at the forefront…
Extensive sweating, or hyperhidrosis, can be an extremely frustrating diagnosis. Although sweating itself is an incredibly normal and healthy process, when it happens excessively, it can impact your day to…
Within 48 hours following her son Kylan's birth, Emily Bussenschutt had already visited three separate hospitals searching for a diagnosis. 9Honey explains that Kylan was born with epidermolysis bullosa…
Cancer is often an avoided topic in conversation - which is understandable. It's scary and uncomfortable to think about, so we like to push it away. And for adolescents and…
Dr. Rachel Grisham MSK ovarian cancer section head discussed clinical trials investigating PARP inhibitors for ovarian cancer patients. The event was a round table discussion and interview for Targeted Oncology.…
Rare Disease Week 2022 Rare Disease Week is the week that leads up to Rare Disease Day, which is recognized on the last day of February. This year, we are…
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…
Over the last few years, many researchers have been utilizing organoids as a pathway to learn more about diseases and spur drug development. Organoids may be helpful, moving forward, in…
According to a story from the Baltimore Sun, a childhood home of Henrietta Lacks - a Black woman often dubbed "the mother of modern medicine" - was unknowingly demolished just…
In the past, treatment for progressive familial intrahepatic cholestasis (PFIC) was symptomatic or surgical, meaning that there weren't too many treatment options for this patient population. This all changed with…
In the past, many researchers have hypothesized a link between multiple sclerosis (MS) and different viruses or viral infections. While these hypotheses are varied in nature, there has rarely been…
Tay-Sachs disease has escaped effective treatment since 1883 when an ophthalmologist names Warren Tay found small red spots in the back of the eyes of some infants. According to an…
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