Orphan Drug designation is granted to therapies that are designed to treat, diagnose, or prevent rare diseases or conditions. In the United States, a rare condition is one affecting fewer…
Nobody expects their child to be diagnosed with cancer. The idea of facing cancer at a young age can be shocking, terrifying. So when 4-year-old Ash Timmerman seemed slightly "off,"…
Raising awareness for rare diseases and conditions is important for many reasons. First, it helps to increase education and understanding around these rare conditions. Awareness also offers support to those…
Caleb Castle-Parker was born ten weeks premature. At the time, his parents didn’t know how his prematurity might affect him (if at all) long-term. But Caleb seemed to be…
According to a story from Healio, in a phase 3 clinical trial, treatment with the drug resmetirom was able to achieve the desired endpoints at 52 weeks in people living…
In late June 2023, global biopharmaceutical company UCB announced that the U.S. Food and Drug Administration (FDA) approved RYSTIGGO (rozanolixizumab-noli) for adults living with AchR or MuSK antibody positive…
Since 2021, biotechnology company Foghorn Therapeutics ("Foghorn") has been developing its therapeutic candidate FHD-286 for a number of malignancies. In one Phase 1 study, researchers are testing the pharmacokinetics,…
Roivant Sciences recently shared updates on the Phase 2b TUSCANY-2 study. Within the study, the team was evaluating RVT-3101 for moderate-to-severe ulcerative colitis. RVT-3101 is a fully human monoclonal…
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…
13-year-old Kaydence Bryant has always had a big personality. This easygoing teen is an honor roll student, loves playing Pokemon and Skyrim, and plays trumpet in the school band.…
Our genes dictate our health in many ways. When working properly, genes encode for the production of various proteins and enzymes throughout the body. So when genes become mutated,…
Do mast cells—a type of white blood cell that plays a part in immune and allergic responses—contribute to the development of eosinophilic esophagitis (EoE)? In the past, eosinophils (another…
According to a story from Healio, a speaker at the Heart in Diabetes CME Conference claimed that postural orthostatic tachycardia syndrome (POTS) is becoming a common complication for long COVID-19…
For the first time ever, people living with severe hemophilia A will have access to a gene therapy solution designed to treat their condition at the source. In a…
There are at least 15 different types of dysautonomia, conditions characterized by autonomic nervous system malfunction. Normally, the autonomic nervous system plays a role in digestion, temperature control, blood pressure…
Aboard the private yacht, the crew and passengers delighted in the sights of the open water. It was relaxing onboard: a welcomed break from the pressures of daily life. The…
In August 2022, Payton Quaife’s doctor recommended running some blood tests. The doctor didn’t want to frighten the family. But Quaife had a number of bruises that were healing poorly…
It is important to improve the treatment landscape for various types of cancer. Currently, there is a wealth of research searching for cancer cures. Until that happens, though, we…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
The Orphan Drug Act of 1983 transformed the rare disease treatment landscape. Prior to the Act’s passage, there was little commercial investment in drug development targeted towards rare conditions.…
Right now, there are no available therapies for people living with Fragile X syndrome (FXS). However, clinical-stage biotechnology company Connecta Therapeutics (“Connecta”) is working to change that. In a recent…
“I never thought that it could happen to me.” Unfortunately, we tend to repeat this refrain when facing unexpected hardships in our life from trauma to illness. For many people,…
Each year, an estimated 21,000 to 143,000 people around the globe die from cholera. This acute diarrheal illness, caused by Vibrio cholerae (V. cholerae) infection, is more common in…
BCMA is a protein that is found in multiple myeloma cancer cells. CAR-T cell therapies such as Carvytki are engineered to home in on BCMA proteins. According to a…
Your HTT gene encodes for the production of huntingtin, a protein that helps your neurons (nerve cells in your brain) function correctly. When mutations happen in this gene, the protein becomes…
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