“Together, we can do hard things.” That’s the mission of Clara Inspired, a nonprofit organization that aims to raise awareness and funds to cure genetic disorders like STXBP1 disorders. It’s…
In an investor news release from biopharmaceutical company Avidity Biosciences, Inc., the company shared that the FDA placed a partial clinical hold on new enrollment for the Phase 1/2…
Previously, the American Kidney Fund launched the Unknown Causes of Kidney Disease (UCKD) Project. Through the project, the AKF is working to better understand how undiagnosed or misdiagnosed causes…
A code for an autism-linked syndrome has now been established and entered into the U.S. diagnostic manual that catalogs health conditions. According to a recent report in Market Watch,…
According to a story from Cancer Network, the results of a recent retrospective study have found that a two-part combination treatment for diffuse large B-cell lymphoma (DLBCL), a rare cancer,…
According to a story from the Irish Examiner, young people in Ireland are encouraged to watch out for signs of meningitis following an abnormal cluster of fatalities and serious illness…
Before you read on, don't forget to take a look at Part 1 of our interview. In Part 1, Savanah discussed what metastatic breast cancer (MBC) is, her diagnostic journey, and treatment…
Aduhelm was recently approved by the FDA for the treatment of Alzheimer’s disease (AD). The FDA approval, the first in the history of the disease, was based on the drug’s…
If you’re into electronic music, you need to check out DJ Funk Puppy. This ten-year-old (who goes by Richard Tyler Greene III in his day-to-day life) is a talented DJ,…
Chances are, you’ll never meet anyone like Lizanne, a 22-year-old woman from the Netherlands. Not only is she extraordinarily passionate about storytelling and filmmaking, but she was diagnosed with a…
When Branden was just a young child, he was diagnosed with a rare genetic disorder called chronic granulomatous disease (CGD). Now, at 33 years old, Branden is passionate about raising…
October is Breast Cancer Awareness Month. This month is dedicated to raising awareness about the impact of breast cancer, raising funds for research, supporting those with breast cancer, and advocating…
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…
In the past, myofibers (terminally differentiated post-mitotic cells that help make up muscle tissue) have been implicated in the development and progression of Duchenne muscular dystrophy (DMD). Basically, researchers have…
In mid-September 2022, specialty pharmaceutical company Acasti Pharma Inc. (“Acasti”) shared via a news release that it had initiated a pharmacokinetic study on GTX-102. Within the study, Acasti hopes…
Families caring for medically complex children face almost unfathomable decisions, often with no prior experience to guide their thinking and acting. Second only to the clinician expertise on which…
At the start of October 2022, biopharmaceutical company Mustang Bio, Inc. shared via news release that the first patient was treated in a study of MB-106. Within the study,…
It’s no secret that receiving a rare disease diagnosis can take excess amounts of time. Various articles share that, on average, it takes anywhere from four to nine years for…
Right now, there are limited pharmacological therapies available for people with nonalcoholic steatohepatitis (NASH). Rather, treatment options focus on healthy diet and exercise, weight loss, alcohol avoidance, lower cholesterol, and…
According to a story from BioPharma Dive, KalVista Pharmaceuticals has halted its clinical trial evaluating an investigational therapy in development for hereditary angioedema. The trial had included a total of…
Did you know that October is Spina Bifida Awareness Month? This month is designed to celebrate those living with spina bifida, spread awareness and education among the general public,…
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…
The joy of being told you have a normal healthy newborn can at times be overshadowed by concern about whether the baby has a yet-to-be-discovered disease. Medical technology has helped…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is…
According to a September 2022 article from OncLive, the European Medicines Agency (EMA) granted Orphan Drug designation to CAN-2409. This therapy is being developed as a potential therapeutic option for…
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