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…
Research is constantly being done on possible treatment strategies for different forms of cancer; there are many patient populations that face an unmet need. Currently, a team of researchers from…
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…
Soleo Health has just announced that they have been provided access to lumasiran by Alnylam Pharmaceuticals. This access will allow the company to administer the treatment to patients diagnosed with…
According to a recent article, a new study shows that by increasing your alcohol intake, you may actually be increasing your risk of head and neck cancer. Head and Neck…
It's officially Rare Disease Week, which is being celebrated from February 22nd until March 2nd. Every year, we take this week to to recognize, support, raise awareness, and advocate for…
In a first-in-human, dose-escalation Phase 1 clinical trial, researchers are evaluating cosibelimab (CK-301) as a potential therapeutic option for those with metastatic cutaneous squamous cell carcinoma (cSCC). So far, shares Cancer…
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