In 2021, Alyssa, who is now thirteen years old, and her family were told that she had acute lymphoblastic leukemia. Alyssa did not respond to either the standard leukemia…
According to a story from AJMC, a recent critical review evaluated the class of therapies known as Bruton tyrosine kinase (BTK) inhibitors and their use in the treatment of Waldenström's…
The American Society of Nephrology held its annual Kidney Week from November 3-6, 2022. During this week, multiple stakeholders within the nephrology sphere met to discuss trends, clinical care…
Currently, the standards-of-care for advanced/metastatic (stage III or IV) melanoma include immunotherapy, targeted therapy, and radiation. Some patients are given anti-PD-1 inhibitors. However, only around 20-30% of those living…
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 press release published on globalgenes.org, the US Food and Drug Administration (FDA) has expanded the label for Oxlumo, a drug developed by Alnylam Pharmaceuticals as a treatment…
At the moment, there are two FDA-approved treatment options for people with idiopathic pulmonary fibrosis (IPF). However, given the needs of patients, it is necessary to develop additional therapeutic…
In a mid-November 2022 press release from biopharmaceutical company CANbridge Pharmaceuticals, Inc. (“CANbridge”), the company shared that its therapeutic option CAN106, which binds to and neutralizes C5 in the…
Rare diseases are, by their nature, rare—and because of this, it can be difficult to spur drug development. The FDA worked to overcome this through the creation of the…
Clinical trials are a crucial part of learning more about certain diseases, as well as better understanding the impact of various therapeutic options. For example, a clinical trial can…
Before you read, don't forget to check out Part 1 of our interview! In Part 1, we discuss Ronya's story, genetic counseling, and why she chose to launch DNA in Color.…
“I spent the majority of my life fearing the weight of VHL will eventually crush me, but thanks to this drug trial, not only do the rest of my fellow…
According to a recent article featured in Biospace, researchers found substantial evidence of a biochemical correction after treating an eleven-year girl for Gaucher Disease. This is the first pediatric case…
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…
According to a story from PR Newswire, the pharmaceutical company Saol Therapeutics has recently announced that it has completed its enrollment goals for its phase 3 clinical trial. This trial…
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 a…
I believe very strongly in the power of thoughts and words as it relates to my health. I say this because I have lived with a painful chronic neurological movement…
The current standards-of-care for those with Duchenne muscular dystrophy (DMD) include corticosteroids, immunosuppressive treatments, physical therapy, and occupational therapy (among others). Gene therapy has the potential to change the treatment…
Editor's Note: Chronic conditions and rare diseases don't discriminate, Patient Worthy and its partners are interested in amplifying the voices of those from all identities and backgrounds. If you have…
Parents and doctors were concerned about Chuck Goodman's health from an early age. In fact, they thought he had a deadly case of childhood leukemia. At around age five, Chuck…
On December 8, 2022, the Rare Disease Legislative Advocates (RDLA) hosted their monthly webinar. These webinar help provide updates to the rare disease community on legislation and other policy initiatives…
Hypoxic ischemic encephalopathy (HIE) is a type of brain dysfunction or brain injury that occurs when the brain is deprived of oxygen during fetal or neonatal stages. Treatment options are…
Unfortunately, life has not always been kind to Zahid Bashir and Sobia Qureshi. The couple lost two children to Pompe disease, a rare genetic disease caused by GAA gene mutations.…
Those living with diseases like Pompe disease and McArdle disease have genetic mutations which prevent their bodies from adequately breaking down glycogen, a type of complex sugar found at…
Researchers at Brown University, in collaboration with an MIT research team, discovered a method of penetrating the blood-brain barrier (BBB) in mice and delivering medication to treat lethal tumors…
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